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

Japan’s Nippon Shinyaku will acquire European rights for a Duchenne muscular dystrophy disease therapy developed by Capricor ...

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

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

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

Cases of atrial arrhythmia in patients with Duchenne muscular dystrophy (DMD) are common but lack a standard solution, and ...

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 ...

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