Sarepta Therapeutics recently demanded a prominent patient advocacy organization censor a video that contained pointed criticism of the company, STAT has learned.

Suneel Ram lives with Duchenne muscular dystrophy, which necessitates a wide variety of medical supports and continual lifestyle adjustments. Duchenne, the most common form of childhood muscular ...

Duchenne muscular dystrophy (DMD) is an X chromosome-linked lethal muscular disorder with progressing muscle wasting and weakness caused by mutations in the gene encoding a subsarcolemmal protein ...

T he Food and Drug Administration on Thursday approved Elevidys, Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy, for nearly all patients with the fatal muscle-wasting disease ...

This new gene therapy treatment has a Minnesota family optimistic 02:22. An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

A young boy died due to a cardiac arrest in a mid-stage trial that tested Pfizer's experimental gene therapy for a muscle-wasting disorder called Duchenne muscular dystrophy (DMD), the drugmaker ...

On Jan. 8, 2020, as I was parking my car, I got a long-awaited phone call from one of my son’s doctors. She informed me that our 7-month-old son, Eliot, had Duchenne muscular dystrophy, a fatal ...