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 ...

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 ...

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

As Western Pennsylvania natives, my wife, Wendy, and I are both Steelers fans. Naturally, when the Steelers played the Dallas Cowboys a couple weeks ago, we tuned in. If you’re not a football fan, you ...

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, ...