News

The FSH Society, a non-profit organization led by facioscapulohumeral muscular dystrophy (FSHD) patients in support of research and education, joined other FSH groups worldwide in hosting the first World FSHD Day on June 20. The day was developed with FSHD Champions, an international alliance of organizations advocating for awareness…

Santhera Pharmaceuticals reports that the European Medicines Agency (EMA) has validated its Marketing Authorization Application (MAA) for its drug Raxone (idebenone;  U.S. brand name, Catena) as a treatment for Duchenne muscular dystrophy (DMD) in patients with respiratory function decline and who are not under glucocorticoid therapy. The EMA validation confirms…

  Raul Carranza was born in Chula Vista 27 years ago. The recent graduate in political science from the University of California San Diego, and future law student,  is one of the university’s most heralded students. Diagnosed with muscular dystrophy (MD) at the age of two, and by his third birthday…

New research suggests that heart arrhythmia in myotonic dystrophy (DM) patients is caused by mutant RNA in the heart’s sodium channel. The study, which adds to the understanding of this symptom, was titled “Splicing misregulation of SCN5A contributes to cardiac-conduction delay and heart arrhythmia in myotonic dystrophy,” and published in …

CureDuchenne, a nonprofit group working to advance research and improve care for people with Duchenne muscular dystrophy (DMD), is partnering with Catabasis Pharmaceuticals to host a webinar discussing promising results from the first part of two-part MoveDMD clinical trial. The webinar is set for Wednesday, June 22, at 1 p.m. ET…

Northbrook, Illinois-based Marathon Pharmaceuticals reports it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the corticosteroid drug deflazacort as a treatment for Duchenne muscular dystrophy (DMD). There is currently no FDA-approved treatment or cure for DMD, the most common and severe form…