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

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 ...

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 ...

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

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 ...