Columnist Betty Vertin, watching her son Chance complete his final season, wishes her sons with DMD could've played football, too.
Treatment with an RNA-based therapy approach was found to correct molecular defects in a cell model of myotonic dystrophy ...
A single low dose of the investigational gene therapy BB-301 helped with swallowing for the first two people with oculopharyngeal muscular dystrophy (OPMD) treated in a Phase 1b/2a clinical trial, ...