PPMD Awards Twelfth Clinic Certification as Part of Effort to Ensure People with Duchenne Receive Optimal Care

The Center for Duchenne Muscular Dystrophy at the University of California, Los Angeles…

PPMD Awards Twelfth Clinic Certification as Part of Effort to Ensure People with Duchenne Receive Optimal Care

The Center for Duchenne Muscular Dystrophy at the University of California, Los Angeles…

Solid Biosciences and its subsidiary, Solid GT, announced today that the FDA and the European Commission have granted Orphan Drug designations for the company’s gene therapy candidate, SGT-001,…

PPMD is disappointed to learn that the FDA denied PTC Therapeutics' first appeal of the refuse to file letter issued in February. As we understand it, this is a process and may involve additional appeals. PPMD will be…

Can you believe 2017 is just around the corner? It will be here before you know it and PPMD is busy putting together an exciting list of events that will continue to connect and inform our Duchenne community. It’s already a packed new year!

Check out where your PPMD family plans to be in 2017 and mark your calendars!…

Parent Project Muscular Dystrophy (PPMD) has awarded a $239,000 grant to Dr. Terence Partridge, Professor of Systematic Integrative Biology, Research Center for Genetic Medicine at Children's National Medical Center. This grant will help Dr. Partridge and his team continue their work in understanding exon skipping technology for future trials in…

PPMD is deeply troubled to learn that health insurer Anthem will not be covering Exondys 51, stating the following position:

“The use of eteplirsen is considered investigational and not medically necessary for all indications, including but not limited to the treatment of Duchenne muscular dystrophy.”

This is not an…

PTC Therapeutics, Inc. today announced new data supporting the potential benefit of ataluren in preserving lung function in non-ambulatory people with nonsense mutation Duchenne. PPMD continues to urge the FDA to provide PTC with a full review and advisory committee meeting.…

Catabasis announced that target enrollment of 30 patients has been reached for Part B of the MoveDMD trial, a 12-week trial to assess the safety and efficacy of edasalonexent (CAT-1004) in Duchenne. Edasalonexent is an oral small molecule that the company believes has the potential to be a…

PTC Therapeutics announced that the Proceedings of the National Academy of Sciences (PNAS) has published new results further validating Translarna's™ (ataluren) mechanism of action to promote readthrough of premature stop codons resulting from nonsense mutations in…

Today, Sarepta Therapeutics and Summit announced a license and collaboration agreement, which grants Sarepta the right to commercialize Summit's utrophin modulators in Europe should they receive approval. This includes ezutromid, which is currently in the Phase 2 PhaseOut DMD trial. PPMD will be hosting a community update call with…