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Recent Research News

Parent Project Muscular Dystrophy (PPMD) understands how important it is for the Duchenne 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.

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)

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)

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)