CHMP upheld earlier opinions that Translarna is not of significant benefit to DMD due to nonsense mutations; European ...

So we have to change society. It’s a tall order, but it’s been done over and over again throughout history. It starts with a ...

Hurricanes in the southeastern U.S. prompt columnist Robin Stemple, who has FSHD, to consider his emergency preparedness plan ...

Treatment with an RNA-based therapy approach was found to correct molecular defects in a cell model of myotonic dystrophy ...

Capricor Therapeutics started its rolling submission seeking FDA approval of its DMD therapy deramiocel for heart muscle ...

Columnist Betty Vertin, watching her son Chance complete his final season, wishes her sons with DMD could've played football, too.

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, ...