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 ...
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, ...
Columnist Betty Vertin, who has seven children, tries to organize the chaos in her family's lives, though no week is typical.