An investigational gene therapy for Duchenne muscular dystrophy (DMD), called SRP-9001 micro-dystrophin, was given fast track designation by…
Articles by Marisa Wexler, MS
Oral Puldysa (idebenone), Santhera Pharmaceuticals‘ investigational treatment for breathing problems caused by Duchenne muscular dystrophy (DMD), will…
Santhera Pharmaceuticals announced the signing of two agreements with Rutgers University related to the development of potential gene therapies…
Both PTC Therapeutics and Sarepta Therapeutics expect to continue providing an uninterrupted supply of their Duchenne muscular dystrophy (DMD)…
The Muscular Dystrophy Association (MDA) has formed a Medical Advisory Team to provide guidance related to research and clinical…
Because of the global COVID-19 pandemic, Italfarmaco has paused enrollment of boys with Duchenne muscular dystrophy (DMD)…
The Muscular Dystrophy Association (MDA) is hosting an online Q&A session today to discuss the best ways that people…
Clinical trial data suggest that edasalonexent — a potential Duchenne muscular dystrophy (DMD) therapy — does not affect the immune…
Final results of a Phase 1 clinical trial support the safety of losmapimod and suggest its effectiveness as a…
Sarepta Therapeutics has taken steps to ensure the supply of Vyondys 53 (golodirsen) and Exondys 51…