Months after causing safety concerns that led the FDA to hit a related trial with a clinical hold, a phase 2 trial of ...

Congestive heart failure or irregular heart rhythms (arrhythmias) occur in all people with Duchenne muscular dystrophy by age 18. Chest and back deformities (scoliosis). Enlargement of the calf ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec.

Parent Project Muscular Dystrophy, and whether the drugmaker uses its money to influence, or even muzzle, debate over its Duchenne medicines. It has also bolstered concerns voiced by some parents ...

After months of uncertainty and controversy, the FDA has given approval to Sarepta's Duchenne muscular dystrophy drug – even though it hasn't yet shown any proof it can slow progress of the ...

"Today's approval addresses an urgent unmet medical need and is an important advancement in the treatment of Duchenne muscular dystrophy, a devastating condition with limited treatment options ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy delandistrogene moxeparvovec (Elevidys), Sarepta Therapeutics said in a statement ...

The day Jaxson DeLeon got his Duchenne Muscular Dystrophy diagnosis, his mom didn’t let herself cry. “If you cry, that’s it. You’re not going to stop crying,” Carmen Ramirez remembers ...