New research led by the University of Portsmouth has revealed how Duchenne muscular dystrophy (DMD), best known for causing ...
A 17-year-old Oakland boy with muscular dystrophy, who has been stuck inside his home for a week after his specialized chair ...
An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.
Sarepta Therapeutics shares dropped 20% after a patient's death raises Elevidys safety concerns. Read why I am downgrading ...
Epicrispr Biotechnologies has raised $68m in the initial close of its Series B financing round to commence the clinical trial ...
Whether caused by Duchenne muscular dystrophy, aging or other degenerative conditions, muscle loss can make everyday activities—like walking, climbing stairs or even standing up—a daily struggle.
Decker was given the 2025 MDA Legacy Award, recognizing his efforts to advance gene therapy, scientific research, and the ...
From Tragedy to Opportunity Sarepta’s Elevidys product is an “adeno-associated virus (AAV)-based” gene therapy aiming to treat inherited muscular dystrophy — one of the most common and most severe ...