News
Parent Project Muscular Dystrophy understands how important it is for the Duchenne muscular dystrophy community to have first hand access to this research, important findings, and any other research updates that come along. Because of this, PPMD monitors and posts all current Duchenne research and community updates. You can also find our latest Newsletter and Person of the Month here.
Newsletter
 It's time to connect! Catch up on the latest research and community updates in this month's End Duchenne eNews.
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2016 News
2015 News
- 12/31/15: Last chance to have your donation doubled!
- 12/30/15: Less than 48 hours left!
- 12/28/15: Submit Written Testimony for Sarepta's Upcoming FDA Advisory Committee Meeting
- 12/28/15: "My mom and dad will match your donation!"
- 12/23/15: EMA Publishes Guidelines on the Clinical Investigation of Medicinal Products for Duchenne
- 12/22/15: Please give now and we'll match your gift!
- 12/22/15: BioCentury Article: How BIO and PPMD plan to create a blueprint for patient preference studies
- 12/21/15: A New Dog Model for Drug Development in Duchenne
- 12/18/15: Dystrophin Mutations Affect Not Only Existing Muscle Fibers, but Satellite Cells As Well
- 12/18/15: How You Can Participate in Sarepta's Upcoming FDA Advisory Committee Meeting
- 12/18/15: BioMarin Announces That FDA Has Advised it Will Not Take Action on Drisapersen New Drug Application by PDUFA Date
- 12/17/15: Another matching gift for early-stage research!
- 12/17/15: PPMD Testifies at FDA PDUFA Stakeholder Meeting
- 12/16/15: Webinar Recording: Understanding the Potential of Epicatechin
- 12/16/15: PPMD Advocates Ensure Duchenne Research Pipeline to Benefit from Congress’s “Omnibus Spending Package"
- 12/15/15: "Why this community matters to me"
- 12/15/15: The Drisapersen Ad Comm: Where We Are Today
- 12/14/15: Marathon Expanded Access Program for Deflazacort: FAQs
- 12/11/15:
- 12/01/15: PPMD Houston Roundtable: Session Recordings
- 12/10/15: 12/16 Webinar: Understanding the Potential of Epicatechin
- 12/10/15: PPMD's Pat Furlong Receives FDLI Distinguished Service and Leadership Award
- 12/09/15: BIO and PPMD Launch Initiative to Share Best Practices on Patient Preference Studies
- 12/07/15: Expanding the Duchenne Therapeutic Opportunities by Targeting Myostatin
- 12/4/15: Register for PPMD's 2016 Advocacy Conference in Washington, DC (February 21-23)
- 11/30/15: Every gift doubled--for every single family.
- 11/26/15: Take a moment today
- 11/24/15: A Historic Day for Duchenne
- 11/23/15: November 24: FDA Advisory Committee Meeting - Live Webcast Information
- 11/18/15: The Big Day is Almost Here!
- 11/17/15: FDA Advisory Committee Scenarios
- 11/16/15: End Duchenne eNews: Tackling Duchenne from Every Angle
- 11/12/15: Webinar Recording: Understanding Myostatin Inhibition
- 11/12/15: PPMD Supports Advancing Targeted Therapies for Rare Diseases Act of 2015 (S. 2030)
- 11/05/15: Webinar Recording: Decode Duchenne Genetic Testing Program
- 11/03/15: Action Alert: Help Us Protect Your Wheelchair Technology!
- 10/28/15: Webinar 11/4: Decode Duchenne Genetic Testing Program
- 10/27/15: A Paradigm Shift in Drug Discovery
- 10/22/15: PPMD Submits Results of Patient-Centered Benefit-Risk Assessment Study in Duchenne & Becker to FDA
- 10/20/15: How You Can Participate in the Upcoming FDA Advisory Committee Meetings
- 10/19/15: Ad Comms Update: Group Letter Submitted to FDA
- 10/14/15: Ad Comms Update: An Overwhelming Day
- 10/13/15: PPMD’s PRO Project: Adding more tools to help with upcoming clinical trials in Duchenne
- 10/9/15: PPMD Urges Senate to Move Forward on #Cures2015 Innovation for Healthier Americans Bill
- 10/7/15: Industry partners receive FDA’s Pediatric Rare Disease Designation and YOU helped make it possible!
- 9/30/15: PPMD Launches Next Phase of Genetic Testing Program, Decode Duchenne in collaboration with BioMarin Pharmaceutical, PTC Therapeutics, & Sarepta Therapeutics
- 9/29/15: New Publication Demonstrates Improved Value of Cardiac MRI in Duchenne
- 9/29/15: The Ensuring Access to Clinical Trials Act (EACT) Passes Senate, then House
- 9/28/15: “Hullabaloo in Duchenne”
- 9/25/15: Update of the Duchenne Care Considerations
- 9/25/15: ACTION ALERT: Don't Let Critical Senate Legislation Stall!
- 9/24/15: FDA Advisory Committee Meetings: An Overview of the Process & Ways to be Involved
- 9/18/15: Urge your Representative to Pass the Ensuring Access to Clinical Trials Act of 2015 (H.R. 209)
- 9/15/15: Bennet, Burr, Warren, Hatch Introduce Bill to Help Patients by Facilitating Faster Development of Drugs for Rare Diseases
- 9/14/15: Webinar Recording: What is an Ad Comm?
- 9/4/15: PPMD Receives $30,000 STRIVE Award From PTC Therapeutics
- 8/26/15: Webinar Recording: Getting Involved with Coach to Cure MD!
- 8/12/15: PPMD’s DuchenneConnect to Receive $1.4 Million Funding Award from PCORI
- 8/11/15: What is an Ad Comm?
- 8/11/15: Imperatives for DUCHENNE MD: A simplified guide to care for Duchenne published in PLOS
- 8/7/15: One month away from World Duchenne Awareness Day!
- 8/6/15: PPMD Submits Comments on FDA's Draft Guidance on Duchenne
- 8/5/15: Action ALERT: Urge your Senators to Cosponsor S. 1597, the Patient-Focused Impact Assessment Act
- 8/3/15: Different Together: PPMD Announces the Duchenne Regulatory Science Consortium
- 7/27/15: Changes to Deflazacort Ordering Process (via Masters Pharmaceuticals)
- 7/21/15: PPMD's 2015 Connect Conference Resources & Recordings
- 7/20/15: PPMD Awards $70,000 Grant to Talem Technologies as Part of Robotics Initiative
- 7/13/15: PPMD's Duchenne/Becker Transitions Meeting Recap
- 7/11/15: Download PPMD's 2015 Connect Conference Booklet
- 7/8/15: Action ALERT: Urge Congress to Vote YES on 21st Century Cures Act
- 7/2/15: Revised Duchenne Anesthesia Recommendations
- 6/29/15: FDA Accepts BioMarin's NDA for Drisapersen; Sarepta Completes NDA for Eteplirsen
- 6/24/15: Webinar Recording: PF-06252616 (Anti-Myostatin)
- 6/19/15: Live from PPMD's Connect Conference! Urge your Senators to Cosponsor PFIA (S. 1597) from Home!
- 6/17/15: PPMD's Annual Connect Conference: Streaming Schedule
- 6/12/15: PPMD Names Duke Medicine Certified Duchenne Care Center
- 6/9/15: Breaking News! FDA Releases Draft Guidance for Duchenne
- 6/3/15: Update on the FDA Guidance
- 6/1/15: Upcoming Webinar: Caregivers
- 5/29/15: Webinar Recording: CAT-1004 Clinical Trial
- 5/26/15: SomaLogic Study Published: Another Piece of the Puzzle
- 5/12/15: PPMD’s Connect Series: Bringing Together You & the Experts
- 5/7/15: Newly Updated Vaccination Recommendations
- 5/6/15: PPMD's 2015 Connect Conference Agenda: Now Available!
- 5/5/15: Thank You!
- 4/29/15: The “Let’s Get Them Moving” Workshop
- 4/27/15: BioMarin Completes Rolling NDA Submission to FDA for Drisapersen for Treatment of Duchenne Amenable to Exon 51 Skipping
- 4/21/15: PPMD Awards $148,000 Grant to University of Washington
- 4/17/15: Webinar Recording: FOR-DMD Study
- 4/13/15: We See Strength in Innovation
- 4/3/15: Webinar Recording & Summary: Orthopedic Complications in Duchenne
- 3/21/15: Recapping the FDA-NIH Dystrophin Methodology Workshop
- 3/18/15: PPMD Participating in FDA-NIH Dystrophin Methodology Meeting on Friday, March 20
- 3/17/15: Dollars Destroying Duchenne
- 3/15/15: PPMD's 2015 Connect Conference Registration Open
- 3/14/15: Spring Forward: Bringing the MD-CARE Act to Life
- 3/13/15: What is Epicatechin?
- 3/12/15: Recall Notice for Deflazacort 30mg
- 3/11/15: Urge Congress to Sign Duchenne Funding Letter
- 3/6/15: PPMD Names Stanford Comprehensive Neuromuscular Clinic at Lucile Packard Children’s Hospital a Certified Duchenne Care Center
- 3/4/15: Dystrophin 101 (WEBINAR RECORDING)
- 3/2/15: We Need YOU to Help Us With the Development of the Solid Suit
- 3/2/15: Important New Duchenne Steroid Protocol: Download the "PJ Nicholoff Steroid Protocol"
- 2/25/15: PPMD & FED Award $1 Million Grant to ReveraGen BioPharma
- 2/20/15: Advocacy Matters: Getting Results to End Duchenne (WEBINAR RECORDING)
- 2/16/15: PPMD Awards University of Missouri $50,000 Exploratory Grant to Explore SERCA2A in Duchenne Dog Study
- 2/12/15: Your Action Plan for the Muscular Dystrophies
- 2/6/15: PPMD Names University of Iowa Children’s Hospital a Certified Duchenne Care Center
- 1/29/15: PPMD Awards UCLA $50,000 Exploratory Grant for Evaluation of Exon Skipping Enhancers in Duchenne
- 1/28/15: Get Active in Advocacy
- 1/26/15: FDA Acknowledges the Importance of Benefit-Risk Preferences: Why this Matters to Duchenne
- 1/23/15: Important Vaccinations Recommendations
- 1/22/15: BMS & The Voice of the Patient
- 1/15/15: Meet Our 2015 PPMD Adult Advisory Council
- 1/12/15: PPMD Welcomes New CEO, John Porter, PhD
- 1/9/15: Update on the 2015 Flu
- 1/6/15: Thank You! Strength Happens Together!
2014 News
- 12/29/14: Heart Failure Drugs Slow Deadly Damage in Duchenne, Offering New Hope
- 12/18/14: Now is the time for strength.
- 12/18/14: Webinar Recording: Combination Therapies
- 12/18/14: The Solid Suit: Combining Forces to Enhance Strength
- 12/16/14: PPMD Names Massachusetts General Hospital a Certified Duchenne Care Center
- 12/13/14: Let's Combine Our Efforts
- 12/11/14: PPMD To Collaborate With Catabasis Pharmaceuticals, Inc. On CAT-1004 Development
- 12/8/14: PPMD Calls for Greater Transparency and Accountability of FDA Patient Engagement Efforts
- 12/8/14: PPMD Awards Akashi Therapeutics, Inc. $500,000 Grant
- 12/3/14: ABLE Act Changes Disability Landscape
This just got real!
- 12/3/14: Webinar Recording & Summary: Duchenne Pulmonary Care for Adults
- 12/1/14: Combine therapies. Combine forces.
- 11/25/14: Finding gratitude in the bittersweet
- 11/25/14: PPMD and Santhera Pharmaceuticals Team Up on New Benefit/Risk Study Focused on Pulmonary Therapies
- 11/24/14: 21st Century Cures: A sign that the tide could be turning
- 11/21/14: Help us bring the ABLE Act to the Floor!
- 11/18/14: PPMD Names Baltimore’s Kennedy Krieger Institute a Certified Duchenne Care Center
- 11/15/14: Webinar Recording & Summary: Duchenne Pulmonary Care for Tweens and Teens
- 11/6/14: Webinar Recording & Summary: Duchenne Pulmonary Care for Tweens and Teens
- 11/3/14: Patients Are Waiting...
- 10/27/14: PPMD Submits Letter to FDA Regarding Latest Sarepta Update
- 10/22/14: PPMD-Funded Gene Therapy Study Improves Walking Ability in Muscular Dystrophy
- 10/22/14: Webinar Recording: Efficacy of Catena®/Raxone® (Idebenone) on Respiratory Outcome in Duchenne
- 10/6/14: Calling All Adults with Duchenne/Becker!
- 10/6/14: Webinar Recording: Prosensa Update about Drisapersen & Other Programs
- 10/3/14: Webinar Recording & Summary: Early Pulmonary Care
- 10/1/14: Upcoming Webinar: Efficacy of Catena®/Raxone® (Idebenone) on Respiratory Outcome in Duchenne
- 9/26/14: President Signs MD-CARE Act Amendments Into Law
- 9/24/14: The MD-CARE Act: Reflections & Projections
- 9/23/14: Duchenne Newborn Screening
- 9/18/14: BREAKING NEWS: MD-CARE Act Passed!
- 9/18/14: September 24 Webinar: Early Pulmonary Care
- 9/17/14: PPMD Names Seattle Children's Hospital Certified Duchenne Care Center
- 9/15/14: End Duchenne eNews: Here to Help
- 9/09/14: Action Alert: Tell the Senate to take action on MD-CARE Act!
- 9/09/14: PPMD Welcomes Annie Kennedy as Senior Vice President of Legislation & Public Policy
- 8/18/14: PPMD Submits Cardiac Recommendations to FDA
- 8/12/14: The Breath of Life: New 3-Part Pulmonary Webinar Series
- 8/06/14: Welcoming Drs. Dongsheng Duan & Jill Rafael-Fortney to PPMD's Scientific Advisory Committee
- 7/29/14: Cincinnati Children’s Named Certified Duchenne Care Center By Parent Project Muscular Dystrophy
- 7/28/14: BREAKING NEWS: Houses Passes MD-CARE Act Amendments!
- 7/24/14: Duchenne Cardiac Working Group Recap
- 7/22/14: PPMD's Pat Furlong on BioCentury This Week
- 7/22/14: No Exon Left Behind
- 7/16/14: End Duchenne eNews: Awarding Grants, Recognizing Progress, Welcoming Leadership
- 7/15/14: One Step Closer
- 7/10/14: PPMD President Testifies Before Key Congressional Committee
- 7/09/14: PPMD and NHLBI Co-Sponsor Duchenne Cardiac Working Group
- 7/08/14: Outcomes Meeting: Laying a Foundation for Outcome Measures
- 7/02/14: Conference 20 Recap
- 6/28/14: PPMD Welcomes John Porter!
- 6/25/14: PPMD Submits FDA Draft Guidance on Duchenne
- 6/23/14: Illinois Designates Week of June 23 as Duchenne Awareness Week
- 6/19/14: PPMD Convenes Outcome Measures Meeting
- 6/18/14: House Schedules Critical Markup on MD-CARE Act
- 6/17/14: Parent Project Muscular Dystrophy Names Children's Hospital Colorado a Certified Duchenne Care Center
- 6/16/14: End Duchenne eNews: Time to Connect
- 6/16/14: Webinar Recording: Ataluren (Translarna) Update with PTC
- 6/13/14: DART Therapeutics Continues Clinical Development of HT-100 for Duchenne
- 6/09/14: PPMD Benefit/Risk Study Results Published in Clinical Therapeutics
- 6/04/14: Lend Your Voice to the FDA Draft Guidance on Duchenne to Speed the Development of Therapies
- 6/03/14: Webinar: SMTC1100 Update with Summit PLC
- 6/03/14: Prosensa Announces A Regulatory Path Forward for Drisapersen as a Potential Treatment for Duchenne
- 5/30/14: Congressional Committee That Funds the FDA Recognizes PPMD-Led Efforts on Benefit/Risk, Draft Guidance; Applauds Collaboration with Agency
- 5/30/14: Upcoming Webinar: Ataluren (Translarna) Update with PTC
- 5/29/14: PPMD’s Pat Furlong will represent the Duchenne community on FasterCures Benefit-Risk Advisory Council
- 5/23/14: EMA grants Conditional Approval for Ataluren
- 5/22/14: PPMD Congressional Champions Voice the Need for Action on the MD-CARE Act
- 5/15/14: One More Reason to Register for PPMD's 20th Connect Conference
- 5/15/14: End Duchenne eNews: Strength Happens Together
- 5/13/14: Santhera Announces Successful Outcome of Phase III Study with Catena®/Raxone® in Duchenne
- 5/12/14: 20th Annual Connect Conference Agenda Now Available
- 5/09/14: Unprecedented Collection of Duchenne Experts Developing Guidance for FDA
- 5/07/14: Results of PPMD-Funded Study Prompt Phase III Study of Tadalafil in Duchenne
- 5/07/14: DuchenneConnect 2013 Annual Report: Registry Growth & Expanded Services
- 4/28/14: Sarepta Webinar
- 4/24/14: New Duchenne Therapies in Development
- 4/21/14: At Long Last, A Victory!
- 4/21/14: Sarepta Announces Plans to Submit New Drug Application to FDA for Eteplirsen for the Treatment of Duchenne by Year End 2014
- 4/15/14: End Duchenne eNews: Transforming Duchenne Care
- 4/14/14: A New Care Guide to Give to Your Provider – Imperatives for Duchenne Care
- 3/31/14: Prosensa Update on Drisapersen (webinar)
- 3/25/14: PPMD Awards $100,000 in Exploratory Grants
- 3/20/14: NEW Certified Duchenne Care Center Program
- 3/18/14: End Duchenne eNews: What Do YOU Think?
- 3/14/14: Action Alert: Ensuring Federal Agencies Working on Duchenne Receive Proper Funding
- 3/12/14: Registration for PPMD's 20th Annual Connect Conference is now open
- 3/05/14: Contemporary Cardiac Issues in Duchenne
- 2/26/14: Nutrition, Energy, and Duchenne
- 2/24/14: Take Action Now!
- 2/19/14: DuchenneConnect Receives $970,000 Grant
- 2/15/14: End Duchenne eNews: Raising Our Voices
- 2/11/14: Webinar: How You Can Help PPMD's Advocacy Agenda
- 2/06/14: Finally, Some Good News!
- 2/05/14: Webinar: ACT DMD Update with PTC
- 2/04/14: Register for the 2014 Advocacy Conference (Feb 23-25)
- 1/28/14: FDA Guidance: Representing the Duchenne Community
- 1/27/14: PPMD Funds Kinnect Project
- 1/16/14: End Duchenne eNews: New Year, New Energy
- 1/15/14: Superheroes Descend on Disney!
- 1/15/14: Clinical Trials in Duchenne: A Behind the Scenes Look (Webinar Recording)
- 1/08/14: College Course Offered in Duchenne
- 1/08/14: Understanding Patient Perspective Critical in Benefit-Risk Assessment
- 1/07/14: Your Simple Action Made It Happen
2012-2013 News
- 12/16/13: End Duchenne eNews: Simple Actions, Lasting Impact
- 12/13/13: Duchenne Policy Forum Recap
- 12/09/13: Together We Can Make a Difference
- 12/06/13: Important Updates on the Duchenne Policy Forum
- 12/04/13: Move Duchenne reasearch forward faster with one simple action
- 11/22/13: Webinar Recording: Update on Natural History Data Study
- 11/18/13: End Duchenne eNews: Time to Unite & Rally
- 11/12/13: Facing Disappointment from FDA's Eteplirsen Response
- 11/06/13: Duchenne Policy Forum
- 10/17/13: Decode Duchenne: A Genetic Testing Program
- 10/15/13: End Duchenne eNews: Pushing the FDA to Accelerate Treatments
- 10/10/13: Pushing the FDA to Accelerate Treatments
- 10/07/13: Clinical Trial Expectations
- 10/04/13: WMS Congress: Considering Care
- 10/04/13: WMS Congress: Next Generation Exon-Skipping and Clinical Trial Updates
- 10/03/13: Important Flu Vaccination Updates for 2013-14
- 09/27/13: Northwestern Mutual is New Corporate Partner of Coach To Cure MD
- 09/20/13: GSK and Prosensa Announce Primary Endpoint Not Met in Phase III Study of Drisapersen
- 09/17/13: End Duchenne eNews: Research & Advocacy Efforts Will Tackle Duchenne
- 09/10/13: PPMD Leadership on Patient-Focused Drug Development Nets Inivitation to Present at Two Influential Meetings
- 08/21/13: Prosensa Webinar Replay
- 08/15/13: End Duchenne eNews: August 2013
- 08/14/13: PPMD to Fund $1 Million in Exon Skipping Projects
- 07/31/13: MD-CARE Act: Is your member in a target state or district?
- 07/24/13: Sarepta Therapeutics announces plans to submit New Drug Application to FDA for Eteplirsen - A significant step for the Duchenne community
- 07/17/13: End Duchenne eNews: FDA Continues to Hear Us
- 07/15/13: Connect Conference Recap
- 07/12/13: Update from Congressional Briefing - TAKE ACTION
- 07/12/13: Update from PPMD/FDA Meeting
- 06/21/13: PPMD Urges HHS Secretary to Update Organ Transplantation Policies to Ensure Pediatric Patients Have Access
- 06/19/13: End Duchenne eNews: Appreciating Parents
- 06/13/13: Webinar Replay: Benefit/Risk Survey Results
- 04/30/13: Drisapersen Update webinar with GSK
- 04/30/13: DuchenneConnect Year-End Report
- 04/29/13: New Mobile App: Locate Duchenne Clinical Trials
- 04/22/13: New PPMD White Paper
- 04/22/13: DOD will provide $3.2 million to the Department of Defense Duchenne Muscular Dystrophy Research Program (DMDRP)
- 04/18/13: End Duchenne eNews: A Rich Drug Development Pipeline
- 04/10/13: GSK's Drisapersen Reaches Primary Objective in Study
- 04/08/13: Drug Development Prospects for Duchenne: The Pipeline is rich and deep, with lots of forward momentum
- 04/03/13: Register for the 2013 Annual Connect Conference
- 03/22/13: PPMD Funds Investigator to Build New Muscle from Skin Cells to Treat Duchenne
- 03/21/13: Summit outlines clinical development plans for Utrophin modulator program for Duchenne
- 03/15/13: End Duchenne eNews: Connecting you to Clinical Trials
- 03/13/13: Webinar Recording: Adult Cardiac Care
- 03/07/13: West Coast Connect Meeting
- 03/07/13: PTC receives funds to support Phase 3 confirmatory trials of ataluren
- 03/05/13: Update on MD-CARE Act Reauthorization Efforts
- 03/01/13: PPMD Funds Project to Identify Genetic Changes that Affect the Severity of Duchenne
- 02/28/13: Cardiac webinar: Care for adults living with Duchenne
- 02/19/13: Advocates in Muscular Dystrophy Community Press Congress for Fast Action on Research and Treatment
- 02/19/13: LAST CALL: Risk/Benefit in Duchenne Therapies Survey
- 02/12/13: Action Alert: Help Reauthorization from Home
- 02/10/13: 11-Time Olympic Medalist Ryan Lochte Plunges Into Advocacy with PPMD
- 02/07/13: Finger Pointing Exercise
- 02/07/13: Larry Bell, MD Joins Prosensa as Head of Global Regulatory Affairs
- 02/04/13: A Strong Start to 2013
- 02/01/13: Parent Project Muscular Dystrophy Funds Collection and Analysis of Imaging Data from Eteplirsen Study
- 01/31/13: Prosensa Achieves Orphan Drug Status on its Entire Duchenne Portfolio
- 01/30/13: Webinar Recording: Cardiac Series - LVAD in Duchenne
- 01/29/13: Is Your District Represented?
- 01/21/13: Webinar Recording: Ataluren Update with PTC
- 01/18/13: Parent Project Muscular Dystrophy Grants $175,000 for Biomarkers, Non-Ambulatory Endpoints in Duchenne
- 01/17/13: Sarepta Summary of Outstanding Questions from PPMD Webinar
- 01/17/13: A New Year, a New Message for Congress
- 01/16/13: Minidystrophin gene therapy effective in dogs
- 01/16/13: Cancer drug Tamoxifen may prove useful for Duchenne
- 01/10/13: GSK Update
- 01/03/13: You made it possible!
- 01/03/13: Prosensa to Present at J.P. Morgan Healthcare Conference
- 12/27/12: Help PPMD make twice the impact on important research
- 12/19/12: Vote for PPMD in Houston Marathon’s Run For a Reason Charity Contest
- 12/19/12: Webinar: Ataluren Update with PTC
- 12/18/12: Exon Skipping & Improving Industry Communications
- 12/18/12: Two Moms, Co-Blogging: The Greatest of These
- 12/18/12: Dantrolene May Help Fight Duchenne
- 12/14/12: End Duchenne Gala Recap & Photos
- 12/14/12: Henri Termeer joins Prosensa as Strategic Advisor
- 12/13/12: Support our dreams and your gift will be doubled
- 12/12/12: Reauthorize the MD-CARE Act
- 12/12/12: Orphan Drug Designation for Myostatin Inhibitor for Muscular Dystrophy
- 12/06/12: Help us advance clinical trials - faster
- 12/06/12: EMA Validation of MMA for Conditional Approval of Ataluren
- 12/04/12: Tivorsan in planning stages for Phase I trial of biglycan
- 12/03/12: Pat Furlong to speak at Institute Of Medicine Roundtable
- 12/03/12: Pat Furlong to speak at Institute Of Medicine Roundtable
- 12/01/12: Upcoming Webinar: Summit, SMT C1100
- 11/29/12: Common Drug Reverses Common Effect of Becker Muscular Dystrophy
- 11/28/12: Protein Injection Points to Muscular Dystrophy Treatment
- 11/26/12: Sarepta Enters Into Collaboration for the Development of Additional Exon-Skipping Product for Duchenne
- 11/19/12: Protect Federal Spending on Rare Diseases like Duchenne
- 11/15/12: November End Duchenne eNews
- 11/13/12: Preclinical muscular dystrophy data shows promise
- 11/07/12: Repeat dosing of SMT C1100 for treatment of Duchenne meets endpoints in Phase 1 trial
- 10/25/12: Q&A Webinar with Sarepta
- 10/24/12: Partnering with the FDA to accelerate approvals
- 10/23/12: Prosensa Announces Progress on Exon Skipping Compounds for the Treatment of Duchenne
- 10/16/12: October End Duchenne eNews
- 10/16/12: Full 48-week Phase IIb data from eteplirsen study in Duchenne presented at World Muscle Society
- 10/11/12: PPMD’s Sharon Hesterlee Featured in Nature Magazine
- 10/10/12: Positive Phase 1 trial results for SMT C1100 (Summit)
- 10/05/12: Save the Date: Q&A Webinar with Sarepta
- 10/03/12: Promising News from Sarepta on Exon 51 Skipping Trial
- 10/02/12: PPMD President Pat Furlong receives RARE Champion Award in Advocacy
- 09/27/12: PPMD awards $50,000 to multi-exon skipping project
- 09/27/12: 2012 Coach To Cure MD PSA
- 09/26/12: Implementing PDUFA
- 09/25/12: Calling all mothers - We need your input!
- 09/25/12: PPMD named one of NerdWallet’s Top 6 Healthcare Innovation Nonprofits
- 09/22/12: Early diagnosis makes a difference
- 09/20/12: PPMD Launches Cardiac Webinar Series 9/26/12
- 09/19/12: Prosensa Named a Fierce 15 Biotech Company
- 09/17/12: Parent Project Muscular Dystrophy awards $280,000 for cardiac gene therapy to University of Missouri
- 09/17/12: Dispatch Article about Serepta Trial
- 09/14/12: September End Duchenne eNews
- 09/07/12: AgeView Press names PPMD President Pat Furlong a 'Belle of Steel'
- 09/06/12: Summit's update on their Duchenne drug development strategy
- 09/06/12: The flu vaccine and Duchenne: What you need to know
- 09/05/12: Why should you join our team?
- 09/04/12: Back to School Webinar
- 08/31/12: Interview with Chris Garabedian, President and CEO of Sarepta Therapeutics
- 08/29/12: Low oxygen boosts stem cell survival in Duchenne therapy
- 08/29/12: Tackle Duchenne
- 08/28/12: Open letter from Chris Garabedian, CEO of Sarepta Therapeutics
- 08/20/12: Clinical Trial Update - GSK2402968 (drisapersen) for Exon 51 Skipping
- 08/13/12: End Duchenne eNews: Go for the Gold
- 08/09/12: Beyond Exon 51
- 08/08/12: Standardizing Duchenne care
- 08/07/12: Progress on Duchenne via antisense exon region skipping
- 08/02/12: Pat Furlong named one of the PharmaVOICE 100
- 07/30/12: Expected Ryan Lochte Events & TV Coverage on NBC
- 07/30/12: Request for Applications for the next competitive round of the Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers
- 07/24/12: Sarepta (formerly AVI) announces significant clinical benefit with eteplirsen after 36 weeks in Phase IIb study
- 07/18/12: End Duchenne eNews July 2012
- 07/17/12: Go for the Gold with Ryan Lochte
- 07/12/12: AVI BioPharma changes its name to Sarepta Therapeutics
- 07/10/12: Ataluren Update (PDF)
- 07/10/12: Action Alert! Contact your Senators to help save vital Duchenne programs at the CDC
- 07/10/12: Landmark FDA legislation becomes law
- 07/09/12: NIH and NCATS Accepting Proposals for TRND Program
- 06/27/12: Clinical Trial First to Test Heart Drug Regimen for Duchenne
- 06/25/12: PPMD to Host First Transforming Duchenne Care Meeting
- 06/20/12: AVI BioPharma and PPMD team up for Rock 'n' Roll Seattle Marathon
- 06/20/12: Patients driving direction of new drug research
- 06/15/12: End Duchenne eNews: Time to Connect!
- 06/13/12: PPMD Leads Critical Letter for Rare Disease Community
- 06/12/12: Anti-stress therapy for muscular dystrophy
- 06/12/12: New Cardiomyopathy Study for Duchenne Recruiting in Ohio
- 06/12/12: Summit achieves SMT C1100 Phase 1 dosing milestone (PDF)
- 06/07/12: All Sites Now Recruiting for GSK Exon 51 Skipping Trial
- 05/31/12: Webinar: New approaches to treating inflammation
- 05/25/12: Summit initiates Phase 1 trial of SMT C1100
- 05/25/12: PPMD endorses Senate version of Prescription Drug User Fee Act (PDUFA) after patient voice strengthened
- 05/22/12: Halo raises $1.1 million to expedite phase 2 study of HT-100
- 05/21/12: 2012 Connect Conference: Early Bird Special Extended!
- 05/17/12: Scientists Discover Clues to Muscle Stem Cell Functions
- 05/10/12: We need your voice to help save important Duchenne programs at the CDC!
- 05/08/12: Bradykinin restores left ventricular function, sarcomeric protein phosphorylation and e/nNOs levels in dogs with Duchenne cardiomyopathy
- 05/08/12: End Duchenne eNews - May 2012
- 05/05/12: Researchers develop new muscular dystrophy treatment approach using human stem cells
- 05/03/12: PPMD holds landmark meeting with FDA leaders
- 05/02/12: http://community.parentprojectmd.org/profiles/blogs/results-from-duchenneconnect-study-webinar-follow-up?xg_source=activity
- 05/02/12: Kitchen Table Conversations: Mothers & Daughters
- 04/26/12: Webinar: Results from DuchenneConnect Study
- 04/26/12: AVI BioPharma presenting additional eteplirsen Phase IIb study data
- 04/24/12: SMT C1100 approved to enter Phase I clinical trial
- 04/24/12: Uncle hikes Pacific Crest Trail to end Duchenne
- 04/10/12: Caregivers: The Power of Female Friendship
- 04/02/12: AVI Summary Announcement
- 04/02/12: AVI BioPharma Announces Eteplirsen Meets Primary Endpoint, Demonstrating a Significant Increase in Dystrophin at 24 Weeks
- 03/30/12: Ask Your Representative to Co-Sponsor FAST Act
- 03/29/12: PPMD Endorses FAST Act Legislation to Expedite FDA Review of Life-Saving Therapies
- 03/26/12: NIH Imaging Biomarker Study Now Recruiting
- 03/26/12: Prosensa’s Clinical Development Plan Update
- 03/23/12: AVI Trial Update (PPMD Webinar)
- 03/22/12: West Coast Connect Meeting: Room Block Closing!
- 03/16/12: ImagingDMD MRI Study (DuchenneConnect Webinar)
- 03/15/12: Kitchen Table Conversations - Vulnerability and Resilience
- 03/14/12: Volunteers needed to Dine Out for Duchenne!
- 03/12/12: AVI BioPharma Announces Late-Breaker Oral Presentation of Phase IIb DMD Study at 2012 AAN Annual Meeting in April
- 03/12/12: Webinar on the ImagingDMD MRI Study on March 14
- 03/09/12: The March End Duchenne eNews is here!
- 03/07/12: Reminder: Register Today for 2012 West Coast Connect Meeting
- 03/01/12: AVI BioPharma Announces Fourth Quarter and Full Year 2011 Financial Results and Recent Corporate Developments
- 03/01/12: Safe and Effective Medicines for Children
- 02/29/12: Latest issue of BIO-NMD's Patient Newsletter
- 02/27/12: TAKE ACTION! 2012 Advocacy Conference Action Alert
- 02/27/12: Duchenne Advocates Take to the Hill
- 02/23/12: AVI BioPharma Announces Fourth Quarter and Full Year 2011 Financial Results and Corporate Update Conference Call
- 02/21/12: PPMD Awards $500,000 to Tivorsan Pharmaceuticals
- 02/21/12: PPMD Announces Board Approved FDA Policy
- 02/14/12: Summit Outlines Clinical Trial Plans for its Duchenne Program
- 02/14/12: JumpStart Invests $250,000 in Milo Biotechnology
- 02/13/12: Trailer - A Life Worth Living: Pushing the Limits of Duchenne
- 02/09/12: The February End Duchenne eNews is here!
- 02/06/12: Duchenne and Bone Health: An Interview with Paula Clemens
- 02/02/12: AVI BioPharma to Present Company Overview at the 14th Annual BIO CEO & Investor Conference
- 02/01/12: Parent Project Muscular Dystrophy is a Featured Nonprofit Selected by Webkinz™ Foundation (PDF)
- 01/31/12: Despite Flat Budget, NIH’s TRND Program Tries to Spark Interest in Rare and Neglected Diseases
- 01/28/12: Important Advocacy Webinar: PDUFA Reauthorization
- 01/26/12: GSK's Clinical Development Plan for Duchenne
- 01/25/12: Prosensa Raises €23 Million in New Equity Financing
- 01/18/12: PPMD Awards Stanley Froehner, PhD, $132,322 Grant
- 01/16/12: NORD's letter to Congressman Stearns
- 01/11/12: The January eNewsletter is here!
- 01/11/12: Enzyme function may discover muscular dystrophy therapies
- 01/04/12: AVI Data Safety Monitoring Board Reports No Safety Concerns Identified in Phase IIb Clinical Trial
- 01/04/12: Halo Therapeutics' HT-100 Receives FDA's Orphan Designation for Duchenne
2011 News
- 12/28/11: Make 2012 the year you Conquer the Canyon to End Duchenne!
- 12/28/11: Important Webinar: Understanding Biglycan
- 12/19/11: Summit Secures $1.5M Agreement with Duchenne Organizations (PDF)
- 12/15/11: PPMD Supports Therapeutic Credit
- 12/14/11: For Academics - NeuroNEXT Webinar
- 12/08/11: December eNewsletter
- 12/06/11: Let's make it happen
- 12/02/11: Ataluren Update (PDF)
- 11/29/11: Webinar: Fat Embolism Syndrome Following Mild Trauma in Duchenne
- 11/29/11: Letter to The U.S. Department of Health and Human Services (HHS)
- 11/28/11: SMT C1100 Receives Orphan Drug Status from FDA
- 11/28/11: Important Webinar Reminder
- 11/22/11: Clinical Trial Announcement from GSK for Exon 51 Skipping
- 11/16/11: TRND Program Announces Next Round of Projects
- 11/15/11: AVI Enters Into Collaborations for Development of Two Additional Exon-Skipping Products for Duchenne
- 11/10/11: Children's Hospital and Pfizer team up on Duchenne program
- 11/10/11: Children's Hospital and Pfizer team up on Duchenne program
- 11/08/11: November eNewsletter is Here
- 11/08/11: Prosensa Named Most Innovative European Biotech SME (PDF)
- 11/08/11: Report from World Muscle Society Meeting - Read Pat's ACE-031 blog | Read Sharon's synopsis of multiple presentations
- 11/03/11: DuchenneConnect Direct Access Webinar: Updates in Genetic Testing - Listen online | Read the Q&A
- 11/01/11: PPMD Signs on to Congressional Letter
- 10/31/11: Moms: We Need You!
- 10/26/11: NIH Continues Support to Dr. Justin Fallon and Brown University with $1.33M+ to Advance Biglycan to Clinic for Duchenne
- 10/24/11: Patient advocacy groups turn to open-access publishing to advance research quest
- 10/20/11: Last Chance: Register for Disney Today
- 10/19/11: Biglycan Named One of Top 10 “Hot Space” Projects to Watch
- 10/19/11: Santhera to Present Clinical Data of Catena® in Duchenne at World Muscle Society
- 10/19/11: Save the Date: DuchenneConnect Direct Access Webinar
- 10/18/11: Continuing to Recruit: MRI Study
- 10/18/11: AVI to Present RNA-Based Duchenne Drug Candidate at World Muscle Society
- 10/17/11: Why Animal Research Needs to Improve
- 10/13/11: Parent Project Muscular Dystrophy Receives Grant from NINDS
- 10/12/11: NORD Releases Study on Orphan Drug Approval
- 10/10/11: GSK Study DMD114117 Recruitment Complete
- 10/07/11: October eNewsletter is Here!
- 10/05/11: First NIH-funded Personalized Drug Center in US to Focus on Muscle Disease
- 10/04/11: FDA Office of Orphan Products Development Welcomes New Director
- 9/29/11: DuchenneConnect Direct Access Webinar Series with Dr. Kathryn Wagner
- 9/28/11: Run For Our Sons Heads to Little Rock Marathon in 2012
- 9/27/11: Pat Furlong Featured on WebMD
- 9/26/11: DuchenneConnect Direct Access Webinar Series
- 9/22/11: Coach to Cure MD 2011 TV Ad
- 9/22/11: PPMD Awards End Duchenne Grant to U Penn
- 9/22/11: Funding Opportunity: NIH Director's Transformative Research Awards
- 9/21/11: Coach to Cure MD: September 24 (video)
- 9/20/11: PPMD Announces Three Cardiac-Specific Grants
- 9/15/11: Share Our Video!
- 9/14/11: Nationwide Children’s Hospital Submits Follistatin IND
- 9/13/11: Prosensa Advances Three Exon Skipping Candidates for Duchenne into the Next Development Stage
- 9/12/11: On Your Mark! Get Set! Done?
- 9/9/11: September eNewsletter: ACTION ALERT!
- 9/9/11: NORD Submits Citizen’s Petition to FDA Regarding Orphan Drug Reviews
- 9/7/11: PPMD Applauds FDA Proposals to Encourage Therapies for Rare Diseases
- 9/2/11: Update on Department of Defense Duchenne Grant
- 9/2/11: PTC Therapeutics & Genzyme Announce Restructuring of Collaboration
- 8/24/11: Department of Defense Announces Funding Possibility
- 8/24/11: Santhera Obtains US Patent for Use of Catena® in the Treatment of Muscular Dystrophies
- 8/19/11: NIH Accepting Proposals for 2012 Awards
- 8/15/11: AVI BioPharma Initiates Dosing in Phase 2 Study of Eteplirsen
- 8/12/11: NINDS Guidance on Rigor and Objectivity in Preclinical Research (PDF)
- 8/11/11: August eNewsletter is Here
- 8/10/11: PLoS Currents Muscular Dystrophy Now Open
- 8/2/11: Heart Failure Drugs Preserve Muscle in Muscular Dystrophy
- 8/2/11: DuchenneConnect Needs You!
- 8/1/11: Run For Our Sons: Summer Update
- 7/25/11: AVI BioPharma Announces Exon Skipping Results Published in The Lancet
- 7/21/11: Coach to Cure MD set for Sept 24 Games
- 7/21/11: EMA Recommends Guidelines in Duchenne/Becker
- 7/19/11: PPMD Conference Presenations are live and ready for download
- 7/12/11: Dr. Francis S. Collins Sets Forth NIH’s Vision for Advancing Translational Sciences: Podcast | Commentary
- 7/5/11: Live Streaming from the 2011 Connect Conference
- 7/1/11: The July eNewsletter is Here
- 6/29/11: Baltimore Mayor Proclaims July 8 “End Duchenne Day”
- 6/27/11: New Controllers for the Disabled Debuts, Promises Change
- 6/23/11: Clinical Genetic Aspects of Duchenne and Becker
- 6/23/11: Listen to the AVI BioPharma Webinar
- 6/16/11: Nothing is More Essential
- 6/16/11: ENMC Workshop Report
- 6/14/11: AVI BioPharma Live Webinar 6/20/11
- 6/14/11: Biglycan Treatment Shows Promise in MDX Mouse
- 6/14/11: Duchenne Boys Needed for IGF-1 Study at CCH
- 6/10/11: The June eNewsletter is Here
- 6/9/11: 2011 Connect Conference: Early Bird Special Ending
- 6/8/11: AVI Names New Chief Medical Officer
- 6/7/11: Now Available: Clinical Services Resource
- 6/6/11: AVI BioPharma Live Podcast 6/20/11
- 6/6/11: Contact Your Senators TODAY!
- 6/1/11: Join STIR. End Duchenne.
- 5/31/11: Connect Conference 2011: Early Bird Registration Extended!
- 5/25/11: DuchenneConnect at Work: Helping Families Pursue Genetic Testing
- 5/24/11: Medical Hero Newsletter by CISCRP
- 5/23/11: DuchenneConnect: May 2011 Newsletter
- 5/18/11: Attention Advocates—Contact your House member today!
- 5/16/11: ICE Presents Final Report of 2010
- 5/10/11: Now Recruiting: MRI and Biomarkers for Duchenne Study (ImagingDMD)
- 5/9/11: Santhera Presents Data from 2-Year Open-Label Intervention Study
- 5/6/11: Duchenne Data Published in Leading Scientific Journal - Read the press release (PDF) | Read the published article (PDF)
- 5/5/11: The May eNewsletter is Here
- 5/4/11: Sildenafil/Taladafil Study at Cedars-Sinai Medical Center IRB-Approved
- 5/3/11: One Voice Summit Report Published
- 5/2/11: Genzyme Ataluren Update
- 4/29/11: PPMD Awards Over $40,000 Grant to SomaLogic Study
- 4/28/11: 2011 Connect Conference Agenda is Here!
- 4/27/11: Physiotherapy Basics
- 4/25/11: Partial Clinical Hold Lifted by FDA for GSK2402968
- 4/21/11: ACE-031 Study Terminated: Update From Acceleron and Shire Read Acceleron/Shire’s statement | Read Pat's Blog
- 4/18/11: Scientists identify gene that could hold the key to muscle repair
- 4/14/11: Teen With Duchenne Keeps on Achieving
- 4/12/11: AVI BioPharma to Present Data for RNA-Based Duchenne Drug Candidate
- 4/12/11: GSK2402968 Clinical Development Programme Update
- 4/11/11: Therapeutics for Rare & Neglected Diseases Accepting Proposals
- 4/8/11: The April eNewsletter is Here
- 4/5/11: Phrixus Announces $890,000 in NIH Funding for Duchenne Program (PDF)
- 4/4/11: PPMD Awards $750,000 Grant to Sildenafil/Taladafil Study (PDF)
- 3/31/11: Seeing Duchenne, Understanding Duchenne
- 3/30/11: Ataluren Program Update from Genzyme (PDF)
- 3/30/11: Idebenone as a Novel, Therapeutic Approach for Duchenne (PDF)
- 3/24/11: Results of Prosensa’s Exon-skipping Trial in Duchenne Published in New England Journal of Medicine. Read the Press Release.
- 3/23/11: Workshop Report: Endocrine Aspects of Duchenne (PDF)
- 3/22/11: Download PPMD’s New Duchenne App
- 3/21/11: Drug screen points the way to potential new Duchenne treatments
- 3/16/11: Report from Genetic Diseases of Children Conference
- 3/16/11: GSK Now Recruiting for Exon 51 Clinical Trial in Non-Ambulatory Boys
- 3/11/11: The March eNewsletter is here
- 3/10/11: Steve Wilton Publishes on Novel Compounds for Duchenne (PDF)
- 3/9/11: Last week, PPMD hosted a webinar regarding DELOS with an update from Santhera on this trial. Listen to the webinar and make sure to register with DuchenneConnect to stay abreast of this and other clinical trials in process.
- 3/7/11: AMT receives grant From Dutch parents’ organization for Duchenne Gene Therapy. Read the release.
- 3/4/11: If you missed PPMD President Pat Furlong on NPR’s Talk of the Nation, click here to listen to the full interview. Pat discusses the role social networking has played in rare disease.
- 3/3/11: Check out GBI Research's report, "Musculoskeletal Disorders Therapeutics to 2016", which provides in-depth analysis of unmet needs, drivers and barriers that impact the global musculoskeletal disorders market.
- 3/3/11: Listen to PPMD Pres. Pat Furlong on NPR’s Talk of the Nation, live today at 3pm eastern. Pat will be discussing the role of social media in rare disease. Visit Talk of the Nation online to download today’s show if you can’t listen live.
- 3/1/11: Congratulations to PPMD’s Board Chairman John Killian for winning the 2011 Cash Sweat & Tears Award from the Run Walk Ride Fundraising Council. We can’t think of a more deserving recipient and are so proud of what John continues to accomplish in the fight to end Duchenne. Read the release.
- 3/1/11: Elizabeth Vroom and our friends at Duchenne Parent Project in the Netherlands marked Rare Disease Day by ringing the bell at NYSE Euronext to open trading. Read more.
- 2/28/11: In honor of Rare Disease Day, the FDA has posted an interview with Charlie Riesebeck and his dad, Chuck. Thanks to the FDA for featuring Duchenne on this extremely important day. Watch now. (The sound is temperamental so you may need to turn it up.)
- 2/24/11: Pat Furlong recaps two weeks of extensive traveling and intensive meetings in her latest blog. Read more.
- 2/23/11: Join us March 3 at 2pm eastern for a DELOS Study update from Dr. Richard Finkel. Click here to learn more about this webinar.
- 2/16/11: The National Institutes of Health announce unique opportunity for clinical trainees.Click here to learn more details.
- 2/16/11: Sanofi-aventis to acquire Genzyme. Read the announcement.
- 2/15/11: Whether or not you were able to join us in Washington, DC for our 2011 Advocacy Conference now is the time to make your voice heard. You are just a few "clicks" away from sending your Senators & Representative an email message asking for their support. Please TAKE ACTION now.
- 2/9/11: PPMD’s February newsletter is here, featuring updates on research, including upcoming webinars, and gearing up for next week’s Advocacy Conference. Check it out.
- 2/8/11: PPMD marks ten years of advocacy with One Voice Advocacy Summit. Your advocacy efforts over the last decade have resulted in $192 million in Duchenne-specific federal investment. Learn more; read the release.
- 2/1/11: PPMD awards PTC Therapeutics $250,000 grant to advance drug discovery program to improve heart function in Duchenne & Becker. Read the release.
- 1/26/11: PPMD President Pat Furlong recently returned from a busy month of traveling. Find out what she’s been up to in her latest blog.
- 1/26/11: Congratulations! Stuart W. Peltz, Ph.D., President and CEO of PTC Therapeutics, Inc. has been elected a Fellow of the American Association for the Advancement of Science (AAAS). Read the release.
- 1/25/11: PPMD Board Member Heinreich Meerman (and twenty friends) to run half marathon in Houston this weekend, just after becoming a father again! Congrats and good luck! Read the feature article.
- 1/25/11: PPMD is adding an extra grant cycle this year for its End Duchenne Grant Award opportunity. Click here to learn more about this exciting, additional opportunity to submit your application.
- 1/20/11: Add your voice to ours at PPMD’s One Voice Advocacy Summit next month in Washington, D.C. This is a critical time for us to speak up in our nation’s capital, but we need you to join us. Learn more today.
- 1/19/11: GSK and Prosensa announce start of Phase III study of investigational Duchenne medication. Read the press release.
- 1/14/11: Check out PPMD’s New Year’s wishes and the latest updates in research, advocacy, and care in our January newsletter.
- 1/10/11: Pat Furlong, Research!America’s 2008 Gordon and Llura Gund Leadership Award, is celebrated in Research!America’s newsletter (page 3) for The New Yorker article, “Mother Courage.”
- 1/10/11: AVI to present on Thursday, January 13 at JP Morgan HealthCare Conference. Click here for more information and to view webcast.
Older News
- View Research News: 2011
- 2/23/11: Join us March 3 at 2pm eastern for a DELOS Study update from Dr. Richard Finkel. Click here to learn more about this webinar.
- 2/16/11: Sanofi-aventis to acquire Genzyme. Read the announcement.
- 2/16/11: The National Institutes of Health announce unique opportunity for clinical trainees. Click here to learn more details.
- 2/8/11: PPMD marks ten years of advocacy with One Voice Advocacy Summit. Your advocacy efforts over the last decade have resulted in $192 million in Duchenne-specific federal investment. Learn more; read the release.
- 2/1/11: PPMD awards PTC Therapeutics $250,000 grant to advance drug discovery program to improve heart function in Duchenne & Becker. Read the release.
- 1/26/11: Congratulations! Stuart W. Peltz, Ph.D., President and CEO of PTC Therapeutics, Inc. has been elected a Fellow of the American Association for the Advancement of Science (AAAS). Read the release.
- 1/25/11: PPMD is adding an extra grant cycle this year for its End Duchenne Grant Award opportunity. Click here to learn more about this exciting, additional opportunity to submit your application.
- 1/19/11: GSK and Prosensa announce start of Phase III study of investigational Duchenne medication. Read the press release.
- 1/10/11: AVI to present on Thursday, January 13 at JP Morgan HealthCare Conference. Click here for more information and to view webcast.
- View Research News: 2010
- 12/29/10: We’re so close to our goal to launch the cardiac initiative and just days from the end of the year. Make your tax-deductible donation today, have your gift doubled, and help us heal hearts in 2011! Learn more.
- 12/28/10: Human protein improves muscle function of muscular dystrophy mice. Read more of Dr. Fallon’s report from a study sponsored in part by PPMD.
- 12/28/10: AVI BioPharma provides update on Duchenne muscular dystrophy program. Read the release.
- 12/23/10: Genzyme provides an update on ataluren. Read the report.
- 12/21/10: Dr. Craig McDonald discusses the Six-Minute Walk Test as an outcome measure in Duchenne. Listen to the podcast.
- 12/20/10: Dr. Ryszard Kole from AVI Biopharma and PPMD President Pat Furlong further reflect on recent findings that Duchenne is a stem cell disease. Read the discussion.
- 12/15/10: PPMD Pres Pat Furlong weighs in on recent article about the new mouse model that implicates stem cells.
- 12/14/10: AVI BioPharma names Chris Garabedian Chief Executive Officer. Read the release.
- 12/10/10: PPMD's Scientific Director explains recent article on new mdx mouse model. Learn more.
- 12/1/10: Observational data from one-year follow-up study validate Six-Minute Walk distance as an outcome measure in Duchenne and Becker. Read the release from PTC Therapeutics.
- 11/2/10: Myostatin exon skipping shows promise for a wide range of muscle conditions. Read more.
- 10/19/10: Viagra may help heart effects of Duchenne. News that this community has been following closely, has now peeked national interest. Read the story.
- 10/18/10: GSK outlines approach to delivering advances in the treatment of rare diseases. Read the release.
- 10/15/10: Pivotal data presented at the World Muscle Society Congress suggest Ataluren slows the loss of walking ability in patients with nonsense mutation Duchenne/Becker. Read the release.
- 10/14/10: Kennedy Krieger Institute launches first Phase II Clinical Trial of heart disease treatment for Duchenne. Learn more.
- 10/13/10: Acceleron presents preliminary ACE-031 results from a Phase 1 Multiple Ascending Dose Study in healthy volunteers. Read the release.
- 10/13/10: Prosensa receives £7.5m milestone payment as part of its program with GlaxoSmithKline in Duchenne. Read the release.
- 10/07/10: Nationwide Children’s Hospital posts podcast of This Month in Muscular Dystrophy featuring Dr. Annemieke Aartsma-Rus, speaking on “Progress in Antisense Therapies in Neuromuscular Disorders.” Click here to listen.
- 10/04/10: Institute of Medicine just published a consensus report on Rare Diseases and Orphan Products: Accelerating Research and Development. PPMD President Pat Furlong participated on the Committee. Check out the report brief and then read the full report.
- 09/30/10: PPMD President Pat Furlong discusses the recent AON meeting in D.C. Read her blog.
- 09/30/10: NIH awards Muscular Dystrophy Cooperative Research Center grants. Read the release.
- 09/22/10: The NINDS/FDA Antisense Oligonucleotide (AON) Therapies in Neuromuscular Disease Conference will stream live on September 27 and 28. Click here for more information, including webcast details and a full agenda.
- 09/13/10: If you are the parent of a child with special needs under the age of 21, please take part in a new survey conducted by Northeastern University as part of a research project funded by the NIH.
- 09/09/10: Acceleron announces collaboration with Shire on novel muscle therapeutics for orphan diseases. Read more.
- 08/26/10: A recent article in Slate addresses the difficulties of developing new therapies, and the importance of the words that we all use in talking about therapy development efforts. Read the article.
- 08/25/10: NIH presents new funding opportunity for investigators. Check out these grants with fall application deadlines.
- 08/19/10: BioMarin to Host Conference Call to Discuss Results of Phase 1 Clinical Study BMN 195 for Duchenne Muscular Dystrophy. Learn more.
- 08/19/10: ACE-031 receives orphan designation in the US. Read the release.
- 08/17/10: Santhera obtains European patent for use of Catena/Sovrima in the treatment of muscular dystrophies. Read release.
- 08/13/10: Clinical development program update: A Phase II, double blind, exploratory, parallel-group, placebo controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability, and pharmacokinetics in ambulant subjects with Duchenne. Read more.
- 08/09/10: PPMD awards $600K to Nationwide Children’s Hospital for gene therapy study. Dr. Jerry Mendell leading follistatin gene therapy. Read more.
- 08/04/10: Acceleron Pharma receives FDA fast track designation for ACE-031 for treatment of Duchenne. Read the release.
- 07/20/10: PPMD awards $20,000 grant to Prothelia. Learn more.
- 07/14/10: PPMD announces grants for clinical study sites. The funds will help offset travel costs for participants in Acceleron Pharma’s Phase 2 clinical study of ACE-031. Read more.
- 07/01/10: New peer-reviewed study concludes that Protandim improves markers of oxidative stress and fibrosis in muscular dystrophy mice. Read the release.
- 07/01/10: Exon Skipping and Duchenne Muscular Dystrophy Therapy: Selection of the Most Active U1 snRNA-Antisense Able to Induce Dystrophin Exon 51 Skipping Read more.
- 06/23/10: Prosensa and GlaxoSmithKline initiate development of four additional products under existing alliance in Duchenne. Read the release.
- 06/18/10: A study to assess the pharmacokinetics, safety, and tolerability of GSK2402968 in non-ambulant subjects with Duchenne, is now recruiting. Drug designed to induce skipping of exon 51. More info.
- 06/17/10: How does the FDA make its decisions? Who makes the rules for drug development? What special accommodations are made for rare diseases? PPMD & Cure Duchenne host a two-part live webinar series with FDA officials in July. Click here for more details.
- 06/04/10: FDA and NIH will co-sponsor a meeting titled Antisense Oligonucleotide (AON) Therapies in Neuromuscular Diseases, taking place on September 27 and 28, 2010 in Washington DC. PPMD will participate in this important meeting. Read more.
- 06/03/10: Costa Rica has ordered the country's largest stem cell clinic to stop offering treatment, saying there is no proof that it is effective. Read more.
- 06/02/10: AVI-4658 demonstrates first ever reported generation of greater than 50% dystrophin-positive muscle fibers in a patient following systemic administration in Duchenne. Read the release.
- 05/28/10: PPMD President Pat Furlong discusses yesterday's open conference call with PTC Therapeutics regarding the ataluren trials. Read her latest blog.
- 05/27/10: Watch a video piece from the University of Florida about Dr. Krista Vandenborne who recently received NIH funding for Duchenne research, thanks in part to an End Duchenne Grant from PPMD.
- 05/26/10: A new clinical trial for the investigational compound GSK2402968 (formerly PRO051) was posted to www.clinicaltrials.gov on 5/24. This study will assess the pharmacokinetics, safety, and tolerability of the compound in non-ambulatory boys with Duchenne who have a dystrophin gene mutation amenable to an exon 51 skip. Visit DuchenneConnect for updates.
- 05/24/10: Please join PPMD on our follow-up, open conference call with PTC Therapeutics regarding the ataluren trials on Thursday, May 27 at 5pm eastern. For more information, click here.
- 05/18/10: Check out this important article by PPMD President Pat Furlong and Barbara Godlew, highlighting how advocacy organizations function as a direct conduit to specialized patient populations and how these groups can provide a communication channel to reach thousands of potential clinical trial participants.
- 05/11/10: PPMD will host a follow up call with PTC Therapeutics regarding the ataluren trials on May 27, 2010 at 5pm eastern. For more information, click here.
- 05/07/10: PPMD End Duchenne Grant leads to $7.5 million NIH award for University of Florida’s Krista Vandenborne, P.T., Ph.D. for her work with MRI and MRS technology. Read the release.
- 05/07/10: PPMD's Senior Director of Research, Sharon Hesterlee, has been traveling nonstop the last week to AVI in Seattle, the BIO meeting in Chicago, and New Directions meeting in Ottawa. Read her blog about trends in biotech and the latest on exon skipping.
- 04/22/10: Informative video about the technique of exon-skipping, how it works and how it’s being applied in clinical testing. The technology was developed, in part, by Steve Wilton, a regular presenter at PPMD’s Connect Conference.
- 04/20/10: Responding to concern within the community regarding the potential for an immune response to exon skipping, Annemieke Aartsma and Hidde Ploegh provided their thoughts. Read more.
- 04/16/10: Brenda Wong presented top-line results from the Phase 2b clinical trial of ataluren in patients with nonsense mutation DBMD at the American Academy of Neurology (AAN) annual meeting today. Read the summary.
- 04/09/10: FDA publishes update on Duchenne drug development and regulatory considerations, including findings from a study held during PPMD’s Annual Connect Conference last year.
- 04/08/10: If you missed today’s conference call with John Crowley, listen to it online now. John was very open about his own clinical trial experiences and comments on the recent ataluren trials. This was an incredible hour with a parent who has shared a very similar journey with many of us.
- 04/01/10: PPMD is thrilled to welcome John Crowley, CEO and President of Amicus Therapeutics, inspiration for the movie “Extraordinary Measures,” for a conference call about his own experiences with the clinical trial process on April 8 at 1pm EST.
- 04/01/10: Family friendly version of Care Considerations available from TREAT-NMD. Download here.
- 03/30/10: Prosensa initiates clinical development of PRO044 in an open label phase I/II study. Read more.
- 03/30/10: The American Academy of Neurology’s annual meeting will be April 12-17, 2010. PPMD will be attending and will provide reports to the community. On the agenda, updates and results of clinical trials on ataluren as well as exon skipping. Until then, check out the latest AVI update expected to be discussed at this meeting.
- 03/26/10: PTC Therapeutics has responded to questions you submitted regarding the ataluren trial. Click here to read their summary of answers. Additionally, Genzyme has issued a joint statement with PTC to the community. Read this message.
- 03/25/10: AVI BioPharma announces update on AVI-5038, its PPMO Duchenne drug candidate, to be presented April 14, 2010 at the American Academy of Neurology Annual Meeting. Read more.
- 03/24/10: Spanish translation of the notes from the ataluren call with PTC is now available.
- 03/22/10: Advocacy in Action: Jackson Laboratory receives $1.44m federal grant for Duchenne models. Learn more.
- 03/19/10: In response to some of your questions about the ataluren survey, PPMD has posted FAQs. And if your child took part in the ataluren trial, and you have not participated in the survey, please do so today.
- 03/18/10: Researchers discover chemical that may protect hearts of muscular dystrophy patients. Read more.
- 03/16/10: If your child took part in the ataluren trial, we want to hear from you. Click here to help us gather important data.
- 03/12/10: FDA Officials go to new length to encourage applications for Orphan-Drug Status. Read more.
- 03/08/10: In response to the press release that went out on March 3, 2010 from PTC Therapeutics regarding the ataluren trials, an open conference call was held on 3/5 for parents to have their questions answered by PTC. Click here to read notes compiled from this important open conference call.
- 03/05/10: PPMD President Pat Furlong talks about the ataluren trial results as it relates to the community, in her latest blog.
- 03/03/10: Ataluren results announced. Read this important statement from PPMD President, Pat Furlong.
- 02/18/10: AVI BioPharma receives grants totaling $500,000 to support continuing development of drug candidates to treat Duchenne. Read more.
- 02/09/10: Günter Scheuerbrandt recaps the TREAT-NMD and NIH conference held last November in Belgium. Learn more about the presentations from this important conference in neuromuscular disease. And if you missed PPMD President Pat Furlong’s blog about the conference, check it out.
- TREAT-NMD’s website helps distinguish between the hype of stem cell research and the hope that this science may bring to diseases including Duchenne. (02/05/10)
- Parents: Help in an effort to reduce the time to diagnosis! Take a survey about your first concerns for your child with Duchenne. (01/26/10)
- BioMarin initiates Phase 1 Clinical Study of BMN 195 for Duchenne. (01/26/10)
- Important revision to Part 2 of Care Considerations. Please download this version; it should replace any previous version. Change takes place on page 185 where indicated, with correction on page 189. (01/21/10)
- PBS TV Special Rare disease diagnosis through technology. Read more about this upcoming TV special. (01/20/10)
- PTC Therapeutics announces additional study of ataluren in patients with advanced nonsense mutation Duchenne/Becker. (01/19/10)
- BioMarin initiates Phase 1 Clinical Study of BMN 195 for Duchenne. (01/13/10)
- Research could predict age of onset of heart problems in Becker muscular dystrophy. (01/13/10)
- View Research News: 2009
- Systemic treatment with AVI-4658 demonstrates RNA exon skipping and dystrophin protein expression in Duchenne patients. (12/23/09)
- PTC gets $50M to launch Ataluren, further studies. (12/23/09)
- PTC opens ataluren trial to Non-ambulatory Patients With Nonsense-Mutation-Mediated. Learn more and check out Pat Furlong’s thoughts. (12/11/09)
- CDC strongly recommends that any close family members and caregivers of someone diagnosed with a neuromuscular disease should receive the H1N1 vaccine. (12/10/09)
- Major medical publication supports 6-Minute walk test, funded in part by PPMD. (12/08/09)
- Lancet Neurology publishes CDC’s Care Considerations after three years of vetting and development. Read this critical document, that provides important information regarding the care of your son. (12/04/09)
- What are the correlations between cognitive impairments and late deletions? A recent report published in HMG Advance Access explains. (11/20/09)
- PPMD President Pat Furlong most recent update from Treat-NMD in Brussels. (11/19/09)
- PPMD President Pat Furlong reports from Treat-NMD in Brussels. (11/17/09)
- Latest issue of Nature Biotechnology weighs in on the business of stem cells. (11/17/09)
- Meet The Pediatric Neuromuscular Clinic at MassGeneral Hospital for Children. (11/12/09)
- PPMD President, Pat Furlong, weighs in on stem cell research report from Costa Rica (11/10/09)
- PPMD awards UCLA latest end Duchenne grant. (11/09/09)
- UPPMD hosts conference on “The Endocrine Aspects of Duchenne Muscular Dystrophy” in Florence later this week. Learn more about the agenda. (10/27/09)
- Check out three recently published articles about exon skipping: 1. From Lancet Neurol 2. From The Journal of Gene Medicine 3. From BMC Medical Genetics (10/27/09)
- Exon Skipping Drug Prevents Muscle Wasting and Maintains Muscle Function in Severely Affected, Dystrophin Deficient Mice. Read the press release and article. (10/23/09)
- PPMD adds name to letter to Congress in support of the Therapeutic Discovery Project Tax Credit. (10/22/09)
- National Health Counsel analyzes potential health care reform and breaks down what it may mean for you as a patient. (10/20/09)
- Cure for Duchenne Lies in Paws of Man's Best Friend – Interview with Joe Kornegay (10/16/09)
- MD STARnet Reports Prevelance of Duchenne Among Males Age 5-24 (10/15/09)
- AMT Receives EMEA Orphan Drug Designation for Duchenne Muscular Dystrophy (10/14/09)
- Prosensa and GlaxoSmithKline form alliance to fight Duchenne. Read the official press release. (10/13/09)
- With H1N1 vaccination questions all over the news, you may be confused about what is best for your child. Dr. Brenda Wong of CCHMC weighs in. (10/13/09)
- CDC releases important brochure about H1N1 and what you can do to prevent your family from getting infected (10/08/09)
- TREAT-NMD wraps up promising, successful workshop. Read the press release (10/02/09)
- PPMD President Pat Furlong recounts her recent trip to London for the TREAT-NMD workshop on exon skipping. (09/29/09)
- CDC and PPMD Complete Duchenne Fact Sheet (09/29/09)
- TREAT‐NMD Workshop re: the Development of Antisense Oligonucleotide Therapies for Duchenne. Read the conference outline and agenda. (09/25/09)
- Less is more: therapeutic exon skipping for Duchenne (09/24/09)
- Prosensa’s RNA based therapeutic PRO051 shows favorable results in the first systemic study in patients with Duchenne. (09/22/09)
- Read 5 things you can do to help prevent the spread of H1N1 virus this flu season. (09/17/09)
- Acceleron Pharma’s ACE-031 Increases Lean Body Mass in Phase 1 Single Dose Clinical Trial (09/14/09)
- Article shares findings of a PPMD sponsored workshop on the struggles families deal with when they receive a Duchenne diagnosis (09/11/09)
- PTC Therapeutics Initiates Registration-Directed Phase 3 Trial of Ataluren in Cystic Fibrosis (09/10/09)
- Check out the latest update on Project Catalyst from PTC Therapeutics (09/03/09)
- Prosensa and TREAT-NMD enter into strategic collaboration for PRO-051 clinical trial planning (9/2/2009)
- Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study (08/27/09)
- Prosensa receives notice of allowance for key patent related to lead compound in development for Duchenne (08/17/09)
- Researchers identify new function for protein missing in Duchenne muscular dystrophy (08/04/09)
- Guenter Scheuerbrandt interviews Treat NMD Coordinator, Professor Kate Bushby about clinical trials for exon skipping (07/22/09)
- Annemieke Aartmsa-Rus, Ph.D., explains the latest research strategies in Duchenne. (07/13/09)
- Science Magazine investigates exon skipping and what is coming next in clinical trials. (07/09/09)
- Dr. Jonathan Finder reports on respiratory care in Duchenne patients. (07/06/09)
- Guenter Scheuerbrandt interviews Treat NMD Coordinator, Professor Kate Bushby about clinical trials for exon skipping (06/18/09)
- International Collaborative Effort for DMD (ICE) comes out with Progress report on Clinical Trials (06/12/09)
- The Council of Health Ministers of the EU adopt strategy to implement national plans for rare diseases, including Duchenne. (06/11/09)
- Automated drug screening with contractile muscle tissue engineered from dystrophic myoblasts (06/05/09)
- AVI BioPharma, Inc. receives additional funds to continue research in skipping Exon 50 (06/04/09)
- The American Society of Gene Therapy analyzes enhanced exon skipping (05/28/09)
- New therapy substitutes missing protein in those with muscular dystrophy (05/26/09)
- Dr. Jonathan Finder reports on respiratory care in Duchenne patients. (05/26/09)
- PPMD President and Founder, Pat Furlong, Speaks at 2009 BIO International Convention (05/21/09)
- Science Magazine investigates exon skipping and what is coming next in clinical trials. (05/19/09)
- AVI BioPharma Announces First Quarter 2009 Financial Results and Funding to Accelerate AVI-4658. (05/14/09)
- Research articles discussed at the latest UPPMD meeting: Article 1, Article 2 (05/11/09)
- AVI BioPharma Announces Department of Defense Funding to Accelerate Development of AVI-4658 for Duchenne Muscular Dystrophy (05/08/09)
- Parent Project Muscular Dystrophy Awards Two End Duchenne Grants (05/06/09)
- Ultrasound imaging now possible with a Smartphone. (05/04/09)
- New report on exon skipping from Gunter Scheuerbrandt, the first part of a series on Duchenne research approaches. (04/30/09)
- UNC Wellstone center leads fight against genetic disease (04/28/09)
- PPMD Researchers Dean Burkin, Brad Hodges of Prothelia to have latest findings published in PNAS (04/21/09)
- Read a new interview with TREAT-NMD Coordinator Katie Bushby (04/16/09)
- New therapeutic target discussed in Nature Reviews Drug Discovery (04/15/09)
- New study on the effectiveness of cough augmentation techniques (03/23/09)
- Scientists Harness Exon-Skipping in Large Animal to Successfully Treat Duchenne Muscular Dystrophy (03/15/09)
- Researchers Develop DNA "Patch" For Canine Form of Muscular Dystrophy (03/15/09)
- Check out slides from first ever Asian Conference on Duchenne: Slide 1, Slide 2 (03/10/09)
- NIH launches Challenge Grants with portion of stimulus plan funding (03/09/09)
- President Obama signs stimulus bill, check out how it will affect health related programs (2/23/2009)
- AVI BioPharma Announces Treatment of First Patient in Systemic Clinical Trial of AVI-4658 for Treatment of Duchenne Muscular Dystrophy (02/20/2009)
- While focusing on heart disease, researchers discover new tactic against fatal muscular dystrophy (02/13/2009)
- An update on cough-assistance techniques for pediatric patients with neuromuscular disease. (02/02/2009)
- AVI BioPharma Announces Successful Clinical Trial of AVI4658 for Treatment of Duchenne Muscular Dystrophy by Exon Skipping (01/26/2009)
- PTC Progress Update for Drug Discovery Program to Identify Novel Agents to Treat Duchenne Muscular Dystrophy (DMD) (01/14/2009)
- Parent Project Muscular Dystrophy Awards First End Duchenne Grant: Brown University to Receive Grant Promoting Translational Research in Rare Muscular Disease (01/14/2009)
- View Research News: 2002-2008
Prosensa raises EUR 18 million Series B financing (12/18/2008)
Research Report from PPMD's Scientific Director (12/12/2008)
Exon Skipping and the Brain (12/11/2008)
Stressed out: the skeletal muscle ryanodine receptor as a target of stress (11/20/2008)
Acceleron Initiates Phase 1 Clinical Trial for ACE-031 to Treat Diseases Involving Muscle Loss (11/10/2008)
Sarcospan, a little protein for a big problem (11/10/2008)
Dental Issues and DMD by Elizabeth Vroom of UPPMD (10/14/2008)
Cell penetrating peptide- conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function. (10/9/2008)
Exon Skipping, A Therapy for Duchenne Guenter Scheuerbrandt, PhD interviews Gerard Platenburg, President of Prosensa (10/8/2008)
PTC THERAPEUTICS announces data showing 6 Minute Walk Test consistently measures ambulatory function in patients with Duchenne Muscular Dystrophy (9/30/08)
Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer (9/30/08)
Self-renewal and expansion of single transplanted muscle stem cells (9/23/08)
Duchenne & Anesthesia: An Old Problem Revisited (9/11/08)
Current Treatment of Adult Duchenne (9/11/08)
A Phase I/II trial of MYO-029 in Adult Subjects with Muscular Dystrophy (9/3/08)
PlGFMMP-9expressing cells restore microcirculation and efficacy of cell therapy in aged dystrophic muscle (9/1/08)
Some important information about Glyconutrients (8/25/08)
Looking under every rock: Duchenne and traditional Chinese medicine (8/25/08)
Improvements to Morpholino’s provide sustained expression of Dystrophin (8/22/08)
The Wisdom of Children: Insight into Clinical Studies (8/20/08)
Progress Update for Drug Discovery Program to Identify Novel Treatments for DMD (8/18/08)
Disease-Specific Induced Pluripotent Stem Cells (8/8/08)
PPMD funded projects happening at the University of Pennsylvania (8/6/08)
Update on Need for Haelan/BBI Trial (8/5/08)
Microarray Based Mutation Detection in the Dystrophin Gene (8/1/2008)
BioMarin and Summit plc Sign Worldwide Licensing Agreement for Duchenne Muscular Dystrophy Program (7/23/2008)
The Emotional Effects of Genetic Diseases (7/03/2008)
Long term administration of antisense oligonucleotides into the paraspinal muscles of mdx mice reduces kyphosis (7/01/2008)
Imbalance between pSmad3 and Notch induces CDK inhibitors in old muscle stem cells (6/23/2008)
Interview with Santhera Pharmaceuticals about Phase II trial with SNT-MC17/Idebenone in Duchenne patients (6/19/2008)
IRES-Mediated Translation of Utrophin A Is Enhanced by Glucocorticoid Treatment in Skeletal Muscle Cells (6/18/2008)
Viagra showing promise with treating cardiac problems in Duchenne (6/16/2008)
Increased treatment options for Duchenne and related muscular dystropies (6/9/2008)
Duchenne muscular dystrophy: an old anesthesia problem revisited (6/5/2008)
NIH Report on Translational Research (6/2/2008)
Cell-lineage Regulated Myogenesis for Dystrophin Replacement: a Novel Therapeutic Approach for Treatment of Muscular Dystrophy (6/1/2008)
AVI BioPharma to Present at the American Society of Gene Therapy Meeting (5/30/2008)
Exon skipping in Duchenne muscular dystrophy (5/27/2008)
Report on the Parent Project Muscular Dystrophy Behavior Workshop in Philadelphia, December 2006 (5/21/2008)
Prosensa announces the start of an international multi-center phase I/II clinical study with ‘smart drug’ PRO051 in patients with Duchenne Muscular Dystrophy (5/12/2008)
Update on Research Approaches for a Therapy of Duchenne Muscular Dystrophy (5/2/2008)
PTC Therapeutics Announces Initiation of Phase 2b Registration-Directed Clinical Trial of PTC124 in Duchenne/Becker Muscular Dystrophy (4/23/2008)
Neuropsychiatric Disorders in Males With Duchenne Muscular Dystrophy: Frequency Rate of Attention-Deficit Hyperactivity Disorder (ADHD), Autism Spectrum Disorder, and Obsessive Compulsive Disorder (4/1/2008)New US Orphan Drug Director to Step Up the Pace (3/30/2008)
Duchenne Community Working Together to Establish Guidelines for Optimal Care (3/12/2008)
In Honor of Awareness Week CDC Launches DMD/BMD Page (2/2008)
Lacking Cure, a New Tack on a Muscle Disease (New York Times) (2/2008)
New England Journal- Local Dystrophin Restoration with Antisense Oligonucleotide PRO051 (12/2007)
Abuterol Increase lean body mass in ambulatory boys with Duchenne and Becker (10/2007)
Dystophin Levels as low as 30% are sufficient to avoid DMD (7/2007)
PTC 124 targets genetic disorders caused by nonsense mutations (5/2007)
DMD: Focus on Pharmaceutical and Nutritional Interventions (9/2006)
6th International Roundtable in Monaco June 23, 2007 (6/2007)
Presentations from PPMD's 2007 Annual Conferrence (7/2007)
Clinical Trial offers hope for young men with DMD (10/2007)
Dental Care and DMD (10/2007)
Santhera Report: Encouraging First Data from Phase IIa Clinical Trial with SNT-MC17 in DMD (10/2007)
Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers: The Wellstone Centers website is up and running! (03/24/06)
PT Management of DMD by Laura Case (07/24/06)
Interview by Guenter Schuerbrant with Dr. Thomas Meier of the Santhera company in Switzerland. Santhera currently has Idebenone (CoQ10) in trial and is developing calpain inhibitors for use in DMD. (03/24/06)
Interview by Guenter Schuerbrant with Professor Rudolf Korinthenberg of the children's hospital at the university of Freiburg, the most "loved" clinical specialist for Duchenne in Germany. (03/24/06)
Applied Clinical Trials Magazine: Educating the Public: A Critical, Unmet Need (03/05/06)
PPMD Anti-Inflammatory Conference Executive Summary (02/02/06)
PPMD Anti-Inflammatory Conference Powerpoint (02/02/06)
Will Bennett Fellowship- End of Year 1 Report (02/02/06)
PPMD Announces Successful Completion of Stage 1 of Drug Discovery Collaboration with PTC Therapeutics, Inc. (12/19/05)
Santhera starts European Phase III Study with its Lead Compound SNT-MC17 (idebenone) in Friedreich's Ataxia (12/15/05)
Groundbreaking research unlocks first-of-its-kind clinical trials for young men with Duchenne muscular dystrophy (12/11/05)
Results of PPMD Survey (12/02/05)
UF Doctors Cast Net To Help Muscular Dystrophy Patients (09/14/05)
New Resource for Research on Exon Skipping (08/18/05)
Report from the 4th Round Table conference in Monaco held on January 15,2005 - English (08/02/05)
Report from the 4th Round Table conference in Monaco held on January 15,2005 - Spanish (08/02/05)
Progress Update for PTC Drug Discovery Research Program (05/19/05)
The key references for management of patients with DMD. (04/21/05)
The Stars are Lining UP! (04/21/05)
Scientific Workshops, Conferences, and Committees Reports (04/01/05)
Utah Dystrophinopathy Registry amended consent form (02/17/05)
Estimates of Funding for Various Diseases, Conditions, Research Areas (03/14/05)
Report on DMD from the Department of Health & Human Services (02/23/05)
Progress Update for Drug Discovery Research Program to Discover Novel Agents to Treat DMD (02/15/05)
PTC Therapeutics Receives Orphan Drug Designation for PTC124 for the Treatment of DMD (01/27/05)
Planning steroid dosage trials in DMD (01/17/05)
PTC Therapeutics: Progress Update (12/29/04)
PTC Therapeutics: FAQ (12/29/04)
"Molecular Patches" or Exon Skipping as a Therapy for DMD. (11/15/04)
"Personalized medicine" stakeholder group will address issues at industry, academia, agency interface. (11/15/04)
Steroids and Duchenne Muscular Dystrophy (DMD) (10/12/04)
Third Roundtable Conference Report — Transfer of the Dystrophin Gene (English) (10/12/04)
Third Roundtable Conference Report — Transfer of the Dystrophin Gene (Spanish) (10/12/04)
Santhera Pharmaceuticals AG And Genzyme Corporation Enter Into Option And License Agreement For Discovery Of Therapies For Muscle Diseases (10/12/04)
Information regarding spinal surgery in DMD. (10/12/04)
Deflazacort in DMD: a comparison of two different protocols. (9/07/04)
PTC Therapeutics Awarded Grant From PPMD For Drug Discovery Research (PDF) (9/03/04)
Respiratory Care of the Patient with DMD (PDF) (8/15/04)
Muscular Dystrophy Research & Education Plan for the National Institutes of Health (PDF) (8/15/04)
Streaming Video: Stretching for DMD (requires RealPlayer) (8/09/04)
2004 Annual Conference Powerpoint Presentations Now Available for Download. (8/03/04)
Respiratory Care of the Patient With Neuromuscular Weakness: The (not so) New Paradigm (7/28/04)
General Principles of Management in DMD by Katie Bushby (PDF) (7/28/04)
Steroids — Speaking the Same Language by Katie Bushby (PDF) (7/28/04)
PPMD advocates the critical need to develop standards for care in DMD (6/15/04)
Voluntary Registry survey for DMD/BMD patients with premature stop codons. (6/10/04)
Spanish Translation of the DMD Round Table in Monaco (PDF) (5/22/04)
Creatine is safe, but not effective, in pediatric neuromuscular disease (5/04/04)
Deflazacort Treatment in DMD (PDF) (5/04/04)
Research Approaches towards a Cure for Duchenne Muscular Dystrophy
Research Approaches towards a Cure for Duchenne Muscular Dystrophy (Spanish version)
124th ENMC International Workshop - Treatment of DMD... (4/11/04)
Additional Information on Therapeutic Approaches (4/04/04)
PPMD-Funded/Initiated Respiratory Consensus Document to be Published (3/31/04)
Report on a DMD Round Table Conference in Monaco on January 17-18, 2004 (3/21/04)
Steroid Treatment and the Development of Scoliosis in Males with DMD (3/15/04)
Parent Project Muscular Dystrophy signs historic agreement with biotech... (2/05/04)
Novel Therapies for Duchenne muscular dystrophy (1/07/04)
New Helpful Links — Medicaid Benefits Resource (12/09/03)
New Helpful Links — IEPs (11/04/03)
NIH Guide for Grants and Contracts (10/25/03)
NIH announces new centers to boost muscular dystrophy research (PDF) (10/14/03)
Advances in Duchenne Muscular Dystrophy Gene Therapy (PDF) (10/10/03)
Correlated NOS-Iµ and myf5 expression... (PDF) (10/4/03)
Proteasome Inhibitor (MG-132) Treatment... (PDF) (10/2/03)
Direct Link to Information on sequencing (10/1/03)
Report on the Round Table on Steroids (PDF) (9/30/03)
Gentamicin administration in Duchenne patients with premature stop codon. (9/22/03)
Nutritional Issues for Duchenne Muscular Dystrophy (PDF)
Muscle Regeneration During NOS Manipulation and Deflazacort Treatment (PDF)
French Gene Transfer Study in DMD, BMD Looks Safe, Promising
Converting DMD to BMD: a genetic medicine by Dr. Sue Fletcher (PDF)
Accurate and Affordable Diagnosis of Duchenne Muscular Dystrophy
Attention patients with Limb girdle muscular dystrophy / gamma-sarcoglycan deficiency
Sequencing the Dystrophin Gene (03/23/03)
Survival in Duchenne muscular dystrophy: Improvements in life expectancy since 1967 and the impact of home nocturnal ventilation. (PDF)
High Risk Rheumatic and Musculoskeletal and Skin Diseases Research (PDF) (01/07/03)
Clinical Trials in DMD: CINRG Update (12/02)
National Institutes of Health: ClinicalTrials.gov homepage
Gene Expression Studies in Arthritis and Musculoskeletal and Skin Diseases (12/02/02)
Laura Case Article on Physical Therapy
Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy.
Stem Cell Research Info
Sizing Up Muscular Dystrophy, Peter S. Zammit & Terrance A. Partridge
Task Force Meeting Summary (5/16/02)
Research News from France
Medicaid Waivers (9/02)
National Health Information Center
- View Community News: 2010
- 12/23/10: PPMD President Pat Furlong shares a holiday poem with you.
- 12/21/10: AMAZING! A second matching gift! Read more.
- 12/16/10: Just in time for her birthday, PPMD President Pat Furlong is featured in The New Yorker magazine as a “World Changer.” Congratulations Pat!! Read more.
- 12/14/10: PPMD announces matching gift for cardiac initiative! Have your online donation doubled. Learn more.
- 12/10/10: Your December eNewsletter is here! Help us celebrate our people of the month and learn more about our cardiac initiative. Click here.
- 12/8/10: For the holidays, PPMD has created this short video for you to share on Facebook, Twitter, through email...however you can reach folks. It tells the world what Duchenne is and what it means to have Duchenne, from the people who know the disease best the people in this community. We are One Voice in this fight and we hope you will help share our message.
- 12/6/10: Parent Project Muscular Dystrophy launches cardiac initiative. Learn more about this critical initiative and find out how you can help.
- 11/19/10: Shop Amazon & End Duchenne. This holiday season, when you shop through Amazon, a percentage of your purchase will come directly to PPMD. All you have to do is click here for more instructions. The more people who use this, the higher the percentage of every purchase that supports our fight to end Duchenne. Shop now!
- 11/10/10: PPMD’s November newsletter is available. Help us celebrate clinical trial participants and meet our new Scientific Advisory Committee. Read more!
- 11/9/10: Congratulations to PPMD President Pat Furlong named one of WebMD’s 2010 Health Heroes! Pat is one of six people chosen for being an "extraordinary American" that is "helping families and their children live healthier lives." Read more!
- 10/19/10: Registration for WALT DISNEY WORLD Marathon Weekend ends 10/28. Don’t miss out on this truly magical weekend! Click here for more information.
- 10/07/10: PPMD’s October Newsletter announces site of 2011 Connect Conference and provides a wrap up of Coach To Cure. Read the newsletter.
- 10/06/10: PPMD remembers Bryan Messick. Read Pat Furlong’s blog.
- 09/20/10: What will you be doing on Coach To Cure MD game day?
- 09/10/10: President Pat Furlong reflects on her college days and urges you to get in the game for Coach To Cure MD! Read her blog.
- 09/08/10: Fall is upon us and the kids are returning to school. Make sure you stay informed with the latest Duchenne and PPMD news. Check out the September newsletter, including updates on Coach To Cure MD!
- 09/08/10: Do you have questions about the new health care reform changes that will apply to coverage on or after Sept. 23, 2010? Submit your questions for Health and Human Services Secretary Kathleen Sebelius and check out her online address. Click here for more details.
- 08/17/10: Action Alert! Learn more.
- 08/05/10: PPMD announces major grant awards and gears up for the best Coach To Cure MD event yet. Check out all the details in the August newsletter.
- 07/07/10: Check out PPMD President Pat Furlong’s latest blog about this year’s Annual Connect Conference.
- 06/18/10: Denver Mayor declares June 26, 2010 “End Duchenne Day” in honor of PPMD’s 16th Annual Connect Conference. Read the release.
- 06/15/10: Masters Pharmaceutical strengthens its commitment to ending muscular dystrophy, helps support PPMD’s Connect Conference. Read more.
- 06/15/10: Going to the PPMD Connect Conference in Denver? Looking for something fun to do during your down time? Check out this great list of things to do in the area!
- 06/14/10: Pat Furlong recently returned from Europe for a DRCI meeting. Read her blog about the parents she met on her trip.
- 06/04/10: Check out PPMD’s June newsletter for the latest info on the upcoming Connect Conference, the Ataluren trials, and our summer advocacy initiative. And meet our Person of the Month, Robert Knight! Read more.
- 06/09/10: It’s your last chance to reserve your hotel room for PPMD’s Annual Connect Conference. As of June 10, our block of rooms will no longer be available and our great room rate will be gone. Book your room today and make sure you have registered for this year’s fantastic Connect Conference.
- 05/18/10: The early bird special for PPMD’s Annual Connect Conference has been extended until May 24. Register today! And if you haven’t booked your hotel room, make sure you take advantage of PPMD’s room block at the Denver Marriott City Center. Rooms are filling up quickly don’t miss out on this great room rate!
- 05/14/10: May 15 has snuck up on many of us, so PPMD is extending our early bird special for the Annual Connect Conference to May 24! Make sure you register today to take advantage of this great rate for this amazing conference!
- 05/14/10: Pennsylvania House of Representatives declares 'Duchenne Muscular Dystrophy Awareness Week', May 24. Read the release.
- 04/30/10: Agendas available for PPMD’s Conference in Denver! Download the Connect Conference agenda and the Duchenne Therapeutic Development Meeting agenda, and register today to get your early bird discount.
- 03/15/10: Registration is open for PPMD’s 2010 Annual Connect Conference in Denver, Colorado, June 24-27. Take advantage of our early bird special by registering today!
- 03/09/10: Check out PPMD’s March newsletter, including new marathons for Spring and the latest update on the ataluren trials.
- 02/18/10: Be our Broadway star! Join Run For Our Sons at the New York City Half Marathon on March 21 and earn free tickets to a Broadway show. Click here for more details.
- 02/09/10: PPMD’s February newsletter has arrived. Find out the latest research updates, and mark your calendar for some exciting events. Read more.
- 02/05/10: When we talk about Duchenne, we tend to focus on our sons. But there is a journey that women go on as mothers, daughters, sisters and as carriers. Read PPMD President Pat Furlong’s latest blog, about how Duchenne affects women, not just emotionally but physically.
- View Community News: 2009
- 12/07/09: Print it out and curl up by the fire with your copy of PPMD's December newsletter!
- 11/10/09: It’s been a busy fall. Find out what’s going on in the community with the latest newsletter.
- 10/08/09: PPMD off to a Fall of Victories! Check out the October newsletter.
- 9/29/09: PPMD Wins Vote For A Cause Contest on Intel's Facebook Page. Check out press release.
- 9/28/09: Thank you, thank you, thank you for making Coach To Cure MD a huge success! Read more.
- 08/20/09: Calling All Parents: GAME ON! Head Regional Captain and mom, Rachel Poysky, needs you to join her incredible team of parent volunteers. Read on...
- 08/11/09: Cool down with the August newsletter, hot off the press!
- 07/23/09: Game on! Coach to Cure MD returns September 26. Learn more about this incredible event and share our video with everyone you know.
- 07/22/09: Registration closing for the 2009 ING New York City Marathon. Submit your survey today!
- 07/21/09: Northwestern Mutual Foundation Honors PPMD supporter and friend Chris Messick for his fundraising efforts with a check for $10K for PPMD!
- 07/13/09: Connect Conference wrap up and exciting advocacy project in this month’s newsletter.
- 07/09/09: Advocate in your own backyard! Sign up today for PPMD’s Summer Advocacy Push.
- 06/16/09: Ready, set, go! Check out the June edition of Run For Our Sons newsletter.
- 06/04/09: Hot off the press! Check out our June newsletter.
- 05/19/09: PPMD President, Pat Furlong, on what it means to be “resilient”..
- 05/07/09: Catch the latest PPMD and Duchenne news in our May newsletter.
- 05/06/09: PPMD’s 2009 Annual Connect Conference Agenda is available.
- 05/04/09: Health advocacy organization, Genetic Alliance, features PPMD this week on its website.
- 04/27/09: PPMD President, Pat Furlong, on why words mean something.
- 04/20/09: PPMD launches Run For Our Sons newsletter. Read more.
- 04/15/09: Tell PPMD 15 things about your family that make you smile.
- 04/09/09: Add your signature to the National Health Counsel’s Petition for Change!
- 04/06/09: Read PPMD President Pat Furlong’s latest blog.
- 03/31/09: On Friday, April 3 at 2pm eastern, the U.S. Department of Health and Human Services presents an interactive Webcast on Affordable Health Care for Kids. Learn more.
- 03/30/09: PPMD President, Pat Furlong, welcomes the new head of Pediatric Cardiology at CCHC, Dr. Jeffrey Towbin.
- 03/24/09: NIH's John D. Porter, Ph.D., explains exciting new grant in neuromuscular disease. Learn more.
- 03/18/09: PPMD Founding President Pat Furlong discusses newborn screening in her latest blog.
- 03/13/09: Registration is open for PPMD's Annual Connect Conference in Atlanta, June 25-28!
- 03/13/09: PPMD to host informal family discussion forum in Sacramento, CA on March 28th. view details.
- 03/09/09: PPMD Founder, Pat Furlong, with an update on therapeutic targets. Read her blog.
- 03/05/09: Spring into Action: Check out PPMD’s March newsletter.
- 03/03/09: Catch up with PPMD Founding President, Pat Furlong.
- 02/23/09 The First Asian DMD conference that will take place on February 28th and March 1st in Bangalore, India, will be broadcasted live on the UPPMD website. Please follow the conference here
- 02/18/09: Find out what PPMD’s Founding President, Pat Furlong, has been up to.
- 02/13/09: Make sure you are getting the tax breaks you deserve. Read more.
- 02/09/09: For the latest PPMD updates, check out February's newsletter.
- 02/02/09: Finding caregivers has never been simpler. Visit http://www.care.com/ for a list of caregivers in your area, specific to your needs.
- 1/29/09: Mark your calendars: World Rare Disease Day 2009 learn more...
- 1/26/09: Last Chance to register for the 2009 Advocacy Conference in Washington DC, February 8-10 2009. We need your VOICE to be heard in Washinton register today..
- 1/8/09: The New Year is off to a busy start. Check out the January Newsletter.
- 1/5/09: Attention Parents! The following survey is being conducted by Boston University: Parental Knowledge and Perspectives on Genetic Counseling learn more..
- View Community News: 2008
- 12/26/08: Register today for the 2009 Advocacy Conference in Washington DC, February 8-10 2009. We need your VOICE to be heard in Washinton register today..
- 12/23/08: Parent Project Muscular Dystrophy signs onto letter to Congress urging increased funding for Biotechnology companies to be included in economic stimulus package read more..
- 12/12/08: Just in time for the holidays, it’s your December Newsletter!
- 12/11/08: Run For Our Sons adds new race to its roster! Join us in Atlanta for the ING Georgia Marathon this March. Click here for registration information.
- 11/26/08: Giving Thanks to YOU ...
- 11/11/08: WALT DISNEY WORLD Marathon and 1/2 Marathon Registration re-opened until November 26, 2009! register here...
- 11/10/08: PPMD’s November Newsletter is hot off the virtual press read it here...
- 10/29/08: PPMD’s first Coach to Cure MD event is a huge awareness success! read more...
- 10/23/08: CHOP needs your participation in a study on clinical trials learn more...
- 10/22/08: Get in the game! October 25 is almost here. Support Coach to Cure MD...
- 10/17/08: DisneyWorld Marathon/Half Marathon Registration Deadline is in Less than 2 Weeks! register now...
- 10/9/08: Reauthorization of MD-CARE Act Signed into Law by President read more...
- 10/8/08: Read PPMD’s October Newsletter view now...
- 10/3/08: PPMD Launches Coach to Cure MD read more...
- 9/27/08: Congress passes the MD-CARE Act Reauthorization! read more...
- 9/25/08: Reauthorization Bill Passes the House of Representatives 418-2, Now onto the Senate... read more...
- 9/23/08: House Votes on Reauthorization of MD-CARE Act Advocate Now...
- 9/10/08: It's Game Time!: Read September's Newsletter read now...
- 9/10/08: The next PPMD Roundtable will be in Vancouver, Canada on October 4th register today...
- 9/10/08: The next PPMD Roundtable will be in Vancouver, Canada on October 4th register today
- 9/4/08: NHLBI and partners have produced a go-to guide regarding clinical studies and clinical trials in children go now...
- 8/20/08: It's a Dad Thing: Check out new group on the PPMD Community site go now...
- 8/6/08: Going for the Gold to End Duchenne - Read PPMD's August Newsletter read now...
- 8/6/08: Olympic Swimmer Takes on New Competition -- Duchenne Muscular Dystrophy read more...
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PPMD uses tools like Trust it or Trash it? to be able to produce the most accurate information possible. This is a tool to help you think critically about the quality of health information (including websites, handouts, booklets, etc.). Click on Who said it? When did they say it? and How did they know? to guide you through the process.
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