Bionews, Inc., the parent company of this site, has partnered with the following nonprofit advocacy organizations to help further the mission of ensuring that patients and caregivers are able to get ...

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 ...

I sat in the front row of the football stadium, watching my second-oldest son, Chance, 17, play football. It’s his senior year, and I’ve been front and center at each game. The rows behind me were ...

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, ...