Despite positive trial data, Sarepta Therapeutics has decided to stop the clinical development of SRP-5051 (vesleteplirsen), ...

Columnist Betty Vertin wishes she could take away the pain over the milestones in the teenage years her sons with DMD will miss.

Specific mutations in the DMD gene influence how long patients retain the ability to walk, even when treated with ...

Three weeks ago, my girlfriend, Amanda, and I embarked on a unique journey by co-founding Rebirth Ensemble (RE), an art-making business with a distinct purpose. This partnership is deeply rooted in ...

A new patient group is being enrolled in a Phase 1/2 trial of delpacibart braxlosiran (del-brax), an experimental and potentially disease-modifying therapy for facioscapulohumeral muscular dystrophy ...