October 4, 2018

Jerry Mendell, M.D. Shares Positive Updated Results from Gene Therapy Micro-dystrophin Trial

  PPMD is excited to learn that Dr. Jerry Mendell of Nationwide Children’s Hospital today shared positive results for the fourth patient in Sarepta’s micro-dystrophin trial, indicating positive functional signals for all of the patients….

Learn More

September 25, 2018

Catabasis Initiates Phase 3 PolarisDMD Clinical Trial For Edasalonexent In Duchenne

Catabasis Pharmaceuticals, Inc. announced the initiation of PolarisDMD, the Company’s Phase 3 trial for edasalonexent in Duchenne muscular dystrophy. PolarisDMD clinical trial sites across the United States will open in the coming days for enrollment of the…

Learn More

September 24, 2018

Sarepta Therapeutics Announces Clinical Hold Lifted for its Micro-dystrophin Gene Therapy Program

We are thrilled to learn that the FDA has lifted the clinical hold for Sarepta’s Duchenne micro-dystrophin gene therapy program. Sarepta previously announced on July 25, 2018, that the FDA placed the program on clinical…

Learn More

September 18, 2018

Your Duchenne Registry Data: Researchers Find Different Mutations Have Varying Effects on Progression

As curators of The Duchenne Registry, we are frequently asking you to submit your genetic test report to the Registry. You may often wonder why we need the actual laboratory report, and why we are…

Learn More

September 13, 2018

Genentech/Roche RG6206 Community Letter

  Today, Roche provided the community on their clinical development program of RG6206, an investigational anti-myostatin adnectin that is designed to block the activity of myostatin. Roche also answers questions they have received in light…

Learn More

September 7, 2018

PPMD & Sarepta Partner to Launch Duchenne Outcomes Research Interchange

I am thrilled to announce PPMD’s plan to launch a patient- and physician-reported outcomes data warehouse – the Duchenne Outcomes Research Interchange (the Interchange) – which will combine data from The Duchenne Registry with clinician…

Learn More

August 30, 2018

Gene Repair Technology Restores Dystrophin Expression in Canine Model of Duchenne

We are encouraged to learn that Exonics Therapeutics, Inc., a biotechnology company focused on developing gene editing therapies to treat patients with Duchenne and other neuromuscular diseases, today announced the publication of a preclinical study…

Learn More

August 30, 2018

Pfizer Terminates Domagrozumab (PF-06252616) Clinical Studies for the Treatment of Duchenne

We are deeply disappointed to learn that Pfizer is terminating two ongoing clinical studies evaluating domagrozumab (PF-06252616) for the treatment of Duchenne: a Phase 2 safety and efficacy study (B5161002) and an open-label extension study…

Learn More

August 22, 2018

ReveraGen Announces First Patient Enrollment in International Pivotal Trial of Vamorolone in Duchenne

  ReveraGen BioPharma announced the initiation and first patient enrollment in a double-blind, placebo- and prednisone-controlled clinical trial (VBP15-004) of vamorolone, a first-in-class dissociative steroidal anti-inflammatory, in Duchenne. This VISION-DMD study will enroll approximately 120…

Learn More

August 16, 2018

Wave Life Sciences Receives US Orphan Drug and Rare Pediatric Disease Designations for WVE-210201

  Wave Life Sciences today announced that the FDA has granted both orphan drug designation and rare pediatric disease designation for WVE-210201 for the treatment of Duchenne. These designations represent the significant progress being made…

Learn More