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 ...

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.

What’s a typical week as a caregiver of three sons with Duchenne muscular dystrophy (DMD) like? As a primary caregiver to Max, 18, Rowen, 15, and Charlie, 13, my honest answer is that I wish I knew!

The Critical Path Institute (C-Path) is launching a task force to advance the development of therapies for limb-girdle muscular dystrophy (LGMD) and will lead the efforts of its members to find a new ...

In my four decades of life with limb-girdle muscular dystrophy, I’ve become comfortable educating everyone I meet about my quality of life and the ways all of us living with chronic, rare conditions ...