New research led by the University of Portsmouth has revealed how Duchenne muscular dystrophy (DMD), best known for causing ...

PTC Therapeutics' Translarna loses EU conditional approval, but countries may allow continued use. PTC submitted four FDA ...

PTC Therapeutics said on Friday its inherited progressive muscle-wasting disorder drug will no longer be available for sale in the EU as the European Commission has decided not to renew its ...

Treatment with Spinraza (nusinersen) can help stabilize or improve motor function in adults with SMA, according to a new ...

Wave Life Sciences Ltd. shares promising updates on AATD & DMD treatments. Click here for my updated look at WVE stock ...

Epicrispr Biotechnologies has raised $68m in the initial close of its Series B financing round to commence the clinical trial ...

For millions of people, losing muscle isn't just about weakness; it's about losing independence. Whether caused by Duchenne muscular dystrophy, aging or other degenerative conditions, muscle loss can ...

Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found ...

A 17-year-old Oakland boy with muscular dystrophy, who has been stuck inside his home for a week after his specialized chair ...

The firm will seek accelerated approval for WVE-N531 in DMD amenable to exon 53 skipping based on FDA feedback and Phase II data.

An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

Decker was given the 2025 MDA Legacy Award, recognizing his efforts to advance gene therapy, scientific research, and the ...