Muscular Dystrophy News1h
FDA OKs trial to test gene therapy candidate in LGMD children
The FDA has given the green light to a clinical trial testing ATA-200, a gene therapy for limb-girdle muscular dystrophy ...
Muscular Dystrophy News2d
Sarepta stops development of exon 51-skipping therapy for DMD
Despite positive trial data, Sarepta Therapeutics has decided to stop the clinical development of SRP-5051 (vesleteplirsen), ...
Muscular Dystrophy News6d
Patients with certain mutations lose their walking ability earlier: Study
Specific mutations in the DMD gene influence how long patients retain the ability to walk, even when treated with ...
Muscular Dystrophy News6d
The missed milestones in the teenage years because of DMD
I have three teenage sons — Max, 18, Rowen, 15, and Charlie, 13 — who have Duchenne muscular dystrophy (DMD). They have met several sweet milestones over the years, and I love celebrating them. When ...