Columnist Betty Vertin, whose days often feel overloaded, describes how it feels when her life as a caregiver is recognized and affirmed.

A mouse model study of Precision BioSciences’ gene-editing therapy PBGENE-DMD found it produced long-term functional ...

Sarepta will soon resume shipping Elevidys for ambulatory Duchenne muscular dystrophy patients after a review of safety data ...

Del-zota was granted FDA breakthrough therapy designation for treating DMD, and its developer is on track to seek its regulatory approval.