The young man died of acute liver injury, a known side effect, Sarepta said in a statement. But the company said the “severity” of the patient’s case had not previously been seen with the therapy, ...

New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of ind ...

Long-Term Safety and Tolerability of Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy: Phase 1 to Phase 3 Clinical ... while Duchenne in girls is very rare. Everyone who has Duchenne will ...

A young girl faces an uncertain future after she was diagnosed with an extremely rare and debilitating medical condition which has 'put a timeline on her life'.

Roche to present latest scientific advancements from its neuromuscular portfolio at Muscular Dystrophy Association 2025 conference: Basel Tuesday, March 18, 2025, 11:00 Hrs [IST] ...

Race officials said the 2024 run allowed them to increase their charitable reach by about 18%, donating to various charities ...

She had a fulfilling life as a true volunteer; a plane spotter in WW II, Juvenille Muscular Dystrophy, Boy Scouts, Girl Scouts, along with 50 years as an avid member with her husband ‘Dick’ at ...

Duchenne muscular dystrophy, or DMD ... The disease primarily affects boys, but in rare cases it can affect girls. Muscle weakness is the main symptom. DMD first affects the proximal muscles ...

Gypsy Rose Blanchard, 33, was speaking in Channel 4's new series, Gypsy Rose: Life After Lock Up, following her release from ...

Family facing massive expenses Condition is progressing very quickly READ MORE: Major changes coming to every hospital ...