Kids News

PPMD is proud to share our partnership with the Cooperative International Neuromuscular Research Group (CINRG) to advance the expanded Duchenne Natural History Study (eDNHS) study. Joining PPMD in support of the eDNHS study is Insmed… The post PPMD Partners with Cooperative International Neuromuscular Research Group, Global Sponsors to Advance Expanded Duchenne Natural History Study appeared first on Parent Project Muscular Dystrophy.

On Thursday, February 26, advocates, clinicians, patient leaders, and policymakers came together for a powerful Senate Special Committee on Aging hearing titled “From Regulator to Roadblock: How FDA Bureaucracy Stifles Innovation.” This conversation served as… The post 25 Years Later: Reflecting on the 2001 MD-CARE Act Hearing and Where We Are Now appeared first on Parent Project Muscular Dystrophy.

We are disappointed to learn the news that Roche has made the difficult decision to terminate their Phase 2 SHIELD DMD study evaluating satralizumab for bone health in Duchenne.  According to Roche, this decision was… The post Roche Announces Termination of Phase 2 SHIELD DMD Study appeared first on Parent Project Muscular Dystrophy.

My heart is heavy today following PTC’s decision to withdraw their NDA submission for atalauren (Translarna). PTC submitted their package to FDA in 2024, based on FDA’s commitment to review the totality of evidence for… The post PTC Therapeutics Provides Regulatory Update on Translarna™ (atalauren) appeared first on Parent Project Muscular Dystrophy.

We applaud a major legislative victory for the rare disease and Duchenne and Becker community — one made possible because advocates like you raised your voices, shared your stories, and sustained pressure on lawmakers. With… The post Rare Disease Advocacy Delivers: Major Federal Wins for Duchenne and Becker Community appeared first on Parent Project Muscular Dystrophy.