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 ...

Dr. Laila Bastaki, Head of the Genetic Diseases Center at the Ministry of Health, emphasized the importance of incorporating ...

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

Forge Biologics, a member of Ajinomoto Bio-Pharma Services and leading manufacturer of genetic medicines, today announced it ...

Michelle C. Werner agonized over whether the gene therapy treatment Elevidys was right for her teenage son. But to him, 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 ...

Forge Biologics will help with the manufacturing of adeno-associated viruses (AAVs) in a collaboration with the non-profit ...