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February 7

Upcoming Webinar: Positive Data from Summit's PhaseOUT DMD Ezutromid Clinical Trial

Join PPMD and Action Duchenne as we collaborate to bring you a webinar update on Wednesday, February 7 at 12:00 PM EST on the report this week that Summit has announced positive 24-week interim results from the open-label Phase 2 proof of concept clinical trial, PhaseOut DMD.
January 31, 2018 | Learn more 

Webinar Recording: Italfarmaco

Webinar Recording: Italfarmaco Provides Givinostat Update

PPMD recently hosted a webinar with Italfarmaco to update the community about the company's development of Givinostat and currently enrolling Phase 3 confirmatory study in Duchenne.
January 29, 2018 | Learn more 

News Update

Register Today for PPMD's 2018 Advocacy Conference!

Join us March 4-6 in Washington DC to advocate for Duchenne directly with those who represent you in Congress. This year's conference is packed with events, including an exciting one day meeting called the Duchenne Patient Focused Compass Meeting.
January 24, 2018 | Learn more 

News Update

Updated Care Consideration Guidelines for Duchenne Published

PPMD is proud to have been part of this effort to update the Care Considerations for Duchenne, published today by the CDC in The Lancet Neurology. Since first being published in 2010, significant advances in Duchenne research and care made it evident that an update to these standards of care was long overdue. Later this spring, a family-friendly version of these standards of care will be released.
January 24, 2018 | Learn more 

News Update

First Duchenne Patient Dosed in Microdystrophin Gene Therapy!

One year ago we announced our $2.2 million dollar grant to Dr. Mendell, Dr. Louise Rodino-Klapac (co-PI), and Nationwide Children’s Hospital. A year later, we have the first child dosed an incredible feat in a disease where it feels like everything takes years and years of waiting! Thank you to everyone who continues to support PPMD’s Gene Therapy Initiative, as well as the generous support of Duchenne foundations: Team Joseph, Team Saij, The Fund for Pete’s Sake, Rashad’s family and the Nicholoff family. The child continues to do well, and PPMD will continue to provide updates on this therapy as information becomes available.
January 10, 2018 | Learn more 

News Update

'Patient Experience Data’ Section Added to FDA Reviews

Transforming our lived experiences into data that regulators can utilize is critical. But there has been no formal mechanism for incorporating this patient-experience data into the regulatory reviews & decisions that are so critical to us. So — we carved that pathway. Today’s news is a terrific example of the impact we continue to make together as a unified Duchenne community!
January 9, 2018 | Learn more 

Invest in Gene Therapy

We did it! Because of your commitment & courage!

We set an ambitious fundraising goal for our Gene Therapy Initiative. And once again, you—our incredibly dedicated community—really came through. You not only matched but exceeded our goal! Your gifts will push forward vital work in gene therapy
January 3, 2018 | Learn more 

News Update

How You Can Report Serious Drug Side Effects to the FDA

To ensure that patient communities have a direct mechanism for reporting experiences with FDA approved products, FDA created the MedWatch form. The form which has versions for both clinical providers to report on behalf of a patient and patient community members to self-report, has recently been updated
January 3, 2018 | Learn more 

News Update

What You Need to Know about Duchenne & Viral Gastroenteritis (That Horrible Stomach Bug!)

While viral gastrointestinal (GI) viruses are no fun for anyone, they are especially worrisome for a person living with Duchenne. Learn more about steps you can take if you get one of these viruses.
December 21, 2017 | Learn more 

December 14

Webinar Recording: Understanding Gene Therapy, Part 3 - Galgt2 and Dup2

As part of our ongoing series on Understanding Gene Therapy, PPMD hosted a webinar with Dr. Kevin Flanigan, Director of the Center for Gene Therapy at Nationwide Children’s Hospital.
December 15, 2017 | Learn more 

News Update

Duchenne Receives ICD-10 Code

Over the last two years, PPMD has led an effort to increase the specificity of the ICD-10 codes for the muscular dystrophies. The implementation of these new codes will create a systematic and sizeable impact on the diagnostic, care, research, and outcomes landscapes for our Duchenne community.
December 11, 2017 | Learn more 

Certified Duchenne Care Center

PPMD Designates UCSF Benioff Children's Hospital San Francisco a Certified Duchenne Care Center

Led by clinic director Dr. Jonathan Strober, the Neuromuscular Clinic at UCSF Benioff Children’s Hospital San Francisco specializes in the diagnosis, treatment, and research of disorders affecting the peripheral nerves and muscle.
December 6, 2017 | Learn more 

News Update

Making Duchenne Care and Clinical Trials Better -- Globally

PPMD is proud to announce the formation of a Global Certified Duchenne Care Center Program, offering a path forward for global neuromuscular patient advocacy groups to certify Duchenne Care Centers within their own countries.
December 5, 2017 | Learn more 

Action Alert

Take Action - We Can Still Impact the Tax Reform Bill!

Both the House and Senate have passed tax bills. Since these bill each contain different provisions, they will now need to be ‘conferenced’ before being signed into law. That means, there is still an opportunity for us to reach out to the elected officials who have been elected to represent us — and let them know how the provisions of this bill will impact each of our families.
December 5, 2017 | Take action 

Invest in Gene Therapy

Invest in gene therapy--all gifts matched!

Expanding our Gene Therapy Initiative is vital to our mission of finding a solution for every single person affected by Duchenne. Please help us raise the $400,000 necessary to advance these revolutionary approaches. And remember: your donation will be doubled through the end of the year!
December 4, 2017 | Donate 

Capricor Webinar Recording

[Webinar Recording] Capricor Therapeutics' HOPE-Duchenne Clinical Trial

PPMD and Capricor Therapeutics recently hosted a webinar to learn about the 12-month results from Capricor's HOPE-Duchenne Clinical Trial, as well as the company’s plans for the HOPE-2 Clinical Trial.
December 4, 2017 | Learn more 

Research Update

November Research Update: Gene Therapy, Accelerating Clinical Trials & More

The momentum in Duchenne research has never felt more active and promising. Your support continues to allow us to work on attacking Duchenne from every angle.
November 30, 2017 | Learn more 

Giving Tuesday

#GivingTuesday 24-hour matching gift!

Show your commitment today! Until midnight tonight—any donation you make will be tripled by our anonymous donor. Every gift matters, and your donation goes three times as far in the fight to end Duchenne.
November 28, 2017 | Donate 

News Update

[Webinar Recording] Wave Life Sciences - Stereopurity & Developing Potential Treatments for DMD

PPMD and Wave Life Sciences recently hosted a webinar to provide an overview of Wave’s stereopure exon skipping oligonucleotide programs for the treatment of Duchenne muscular dystrophy and an introduction to the planned Phase 1 clinical trial.
November 17, 2017 | Learn more 

Fibrogen Webinar

[Webinar Recording] FibroGen Update on Pamrevlumab & Enrolling Non-Ambulatory Study

PPMD and FibroGen recently hosted a webinar to hear a community update on FibroGen’s development of Pamrevlumab and currently enrolling FGCL-3019-079 non-ambulatory study in Duchenne.
November 1, 2017 | Learn more 

News Update

Calling All Adults with Duchenne/Becker -- Join the 2018 PPMD Adult Advisory Committee!

PPMD is seeking adult members of our Duchenne/Becker community to serve on the 2018 PPMD Adult Advisory Committee (PAAC). Please consider lending your time, talent, and vision to paving our pathway forward! Apply by Friday, November 17th.
October 30, 2017 | Learn more 

News Update

The Future of Ataluren: A Hard Day But Still a Path Forward

Today the FDA issued a Complete Response Letter regarding PTC Therapeutics’ application for ataluren, noting that “an additional adequate and well-controlled clinical trial(s) will be necessary at a minimum to provide substantial evidence of effectiveness.” This decision is disappointing and not the outcome that our community hoped it would be. However, today’s decision is not the end of the ataluren story. Today’s communication maps out a few paths forward and with those paths come continued hope for an eventual approval of ataluren in the U.S. PPMD will work with PTC, doing what we can to support them while they develop a plan to collect the data the FDA has requested. PTC has also indicated that they plan to file a formal dispute resolution request next week. We will learn more and determine what role this community can play in that process.
October 25, 2017 | Learn more 

Webinar Recording

[Webinar Recording] MoveDMD Trial Open-Label Extension Results

PPMD and Catabasis Pharmaceuticals recently hosted a webinar to hear a community update on the results from the open-label extension of the MoveDMD trial.
October 16, 2017 | Learn more 

News Update

Your DuchenneConnect Registry Data is Advancing Research! New Publication

An article was recently published in Neuromuscular Disorders using data from DuchenneConnect. Dr. Richard Finkel from Nemours Children's Hospital in Orlando, FL led a team of researchers who found that the age at onset of ambulation (age at first steps) is significantly delayed in boys with Duchenne. They suggest that age at onset of ambulation could be a primary outcome measure in clinical trials involving treatment in early infancy.
October 16, 2017 | Learn more 

News Update

Gene Transfer in the Spotlight

Gene transfer -- a technology that PPMD continues to make extensive investments in thanks to your support -- was a highlighted topic at the most recent Wellstone Center meeting hosted by Dr. Eric Olson and his Wellstone team at UT Southwestern Medical Center. A recap of the incredible science presented at this meeting can be found in our latest blog. Thank you to our community for supporting PPMD’s ongoing investment in such innovative technology!
October 11, 2017 | Learn more 

News Update

Steroid Dosing & Duchenne: FAQs

Steroid dosing in Duchenne can be a confusing topic what is enough, what is too much, why weekend doses are higher. Our thanks to Dr. Doug Biggar and Dr. Anne Connolly for helping us address these FAQs for the community. *NOTE: Remember to ALWAYS consult your physician before making any changes to your current dosing regimen.
October 5, 2017 | Learn more 

News Update

[Webinar Q&A] PART 2: Gene Therapy - Nationwide Children's Hospital

Dr. Jerry Mendell of Nationwide Children’s Hospital recently joined us for a webinar to discuss his upcoming gene therapy trial, including trial design, inclusion/exclusion criteria, and timelines.
October 2, 2017 | Learn more 

Coming Full Circle: The Ataluren Ad Comm

Thank you to the families that participated in today’s meeting. Your testimony, as well as the written testimonies of those families who submitted prior to the Ad Comm, had a tremendous effect as indicated by almost every committee member.
September 28, 2017 | Learn more 

News Update

September 28: FDA Advisory Committee Meeting for Ataluren - Live Webcast Information

On Thursday, September 28, an FDA Advisory Committee will review ataluren for potential approval and use within our Duchenne community. A live webcast will be provided starting an 9:00 AM ET so you can follow along from home.
September 27, 2017 | Learn more 

Action Alert

Action Alert: Stop the Graham-Cassidy proposal in the Senate!

The Senate is considering another effort to repeal the Affordable Care Act (ACA). We must urge the Senate to protect people with Duchenne by voting “NO” on the Graham-Cassidy proposal.
September 19, 2017 | Learn more 

2017/2018 Flu Vaccination Recommendations

Flu shots are critical for people with Duchenne -- schedule yours today! Learn what’s new for the flu in 2017/2018, as well as the CDC's recommendations for the flu shot, on our blog.
September 18, 2017 | Learn more 

News Update

[Webinar Recording] PART 2: Gene Therapy - Nationwide Children's Hospital

Dr. Jerry Mendell of Nationwide Children’s Hospital recently joined us for a webinar to discuss his upcoming gene therapy trial, including trial design, inclusion/exclusion criteria, and timelines.
September 14, 2017 | Learn more 

Certified Duchenne Care Center

PPMD Designates Nemours/Alfred I duPont Hospital for Children a Certified Duchenne Care Center

PPMD is excited to announce our 17th Certified Duchenne Care Center -- Nemours/Alfred I duPont Hospital for Children!
September 14, 2017 | Learn more 

MD Community Moves Forward ICD Code Nomination for Duchenne/Becker

The lack of an ICD code specific to Duchenne/Becker has proven a barrier to diagnosis, care, surveillance, research, and access. Today ur global MD community has come together to move forward a nomination for greater specificity in coding. It is our hope that a specific ICD code for Duchenne or Becker MD will allow for payers to better understand how investments into therapeutic interventions can impact cost utilization long-term. We are also eager to be able to assess how care standards are being implemented.
September 13, 2017 | Learn more 

PPMD Lends Leadership to NCATS Rare Disease Research Toolkit Development

PPMD is grateful for the vision, inspiration, dedication, and support of our NCATS partners in developing this Toolkit. It is our hope that this Toolkit will help to facilitate therapeutic development across the research continuum.
September 13, 2017 | Learn more 

News Update

PPMD's 2018 End Duchenne Tour -- Coming to a City Near You!

PPMD is excited to announce next year’s lineup of areas we will visit as part of our End Duchenne Tour!
September 9, 2017 | Learn more 

News Update

How You Can Participate in the Ataluren Ad Comm

The FDA has announced the scheduling of a September 28th Advisory Committee Meeting for PTC’s therapy ataluren. There are multiple ways that Duchenne community members can share their personal experiences with the Advisory Committee reviewing this product.
August 22, 2017 | Learn more 

News Update

Webinar Recording - Gene Therapy for Duchenne

PPMD recently hosted a webinar focused on understanding the different therapies that are being developed that are commonly referred to as gene therapy, including micro-dystrophin and CRISPR/Cas9, how are they similar and different, and what are the challenges and limitations for each of them. If you missed it, the recording of the webinar is now available!
August 15, 2017 | Learn more 

Upcoming Webinar - August 9

Join PPMD's Every Single One Tour in Charleston, WV

It's not too late to join PPMD's Every Single [One] Tour in Charleston, WV on September 9! Combining each of the pillars that make up PPMD's mission -- To End Duchenne -- the Every Single [One] Tour brings free day-long meetings featuring updates on research, advocacy, and care to Duchenne families, professionals, and support networks across the country.
August 10, 2017 | Learn more 

News Update

Newly Published Data Validates Micro-Dystrophin Gene Therapy Approach in Animal Model for Duchenne

At the start of the year, PPMD made its biggest investment ever -- $2.2M to Jerry Mendell at Nationwide Children’s Hospital -- for a Phase 1 Safety trial of gene therapy in Duchenne. This new publication of data showing that the technology works very well in dogs gives us added evidence that gene therapy is ready to go into humans and supports our decision to invest heavily in this approach. While dogs and humans are not exactly the same, these results are so very encouraging. We look forward to working with Dr. Mendell and his team at Nationwide and Sarepta, our co-investors, to move this trial forward.
July 31, 2017 | Learn more 

News Update

PJ Nicholoff Steroid Protocol Published

We pleased to now announce that the PJ Nicholoff Steroid Protocol has now been published in PLOS Currents Muscular Dystrophy. It is our hope that this publication will make it into the reference library of every Duchenne and emergency room provider.
July 18, 2017 | Learn more 

News Update

2017 Connect Conference Resources & Recordings

Check out our online archive of presentations to review some of the incredibly informative panels and discussions from this year's meeting. And mark your calendars -- the 2018 Connect Conference will be held in Scottsdale, Arizona, June 28 - July 1!
July 17, 2017 | Learn more 

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