Sidra Medicine s Genetic and Genomic Medicine clinic is providing life altering gene therapy for rare genetic disorders. In a ...

Duchenne muscular dystrophy is a severe and progressive disorder caused by mutations in the dystrophin (DMD) gene that lead ...

A Phase 3 clinical trial investigating Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular ...

"My medicine is making me stronger," 7-year-old Hudson Sanford says after receiving a breakthrough gene therapy for Duchenne ...

EMA re-confirms non-renewal of conditional marketing authorisation of Duchenne muscular dystrophy medicine, Translarna: Amsterdam, The Netherlands Monday, October 21, 2024, 10:00 ...

SAVANNAH, Ga. -- Delandistrogene moxeparvovec (Elevidys), a gene therapy approved to treat Duchenne muscular dystrophy, ...

DMD is a genetic disease that causes progressive muscle weakness and degeneration caused by a mutation in the dystrophin gene. It’s the most common inherited neuromuscular disease and affects ...

2024 — Researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments for people with ...

showing >5% dystrophin production. The safety profile of WVE-N531 is strong, but competition from gene therapies and the need for further efficacy data present significant challenges. Investors ...

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has once again snubbed Translarna ...