A possible gene therapy approval and a series of major trials readouts set the stage for a busy year of drug development in ...

Six-year-old Cooper Wood was diagnosed with Duchenne muscular dystrophy earlier this year. When his parents applied for a one ...

The embryos were female, and Jenssen remembers the doctors assuring her that, because the Duchenne mutation is linked to the ...

Meet Sehba Shah, Pakistan's inspiring woman chess player who lives with muscular dystrophy yet dreams high above the ...

Neu refix granted orphan disease designation (ODD) & rare pediatric disease designation (RPDD) by US FDA for treatment of ...

The FDA has granted Orphan Drug and Rare Pediatric Disease Designations to Somite’s cell replacement therapy for the ...

A quarter of a million Americans are living with muscular dystrophy or a related neuromuscular disorder. It's the reason ...

ATHENS, Tenn. (WTVC) — Six-year-old Cooper Wood was diagnosed with Duchenne muscular dystrophy earlier this year. When his parents applied for a one-time gene therapy infusion that Cooper is ...

AI-driven biotech Somite.ai scores dual FDA designations for its novel treatment, potentially fast-tracking hope for families ...

To provide hope for families grappling with Duchenne muscular dystrophy across Upstate New York, Buffalo-based Suneel’s Light Foundation has launched a new community grant program, Brighter Days Commu ...

Immunosuppressants used in adeno-associated virus (AAV) gene therapy for monogenic disorders present an adverse effect ...