The evidence for Elevidys is growing, and Sarepta is working to make the DMD treatment widely available, the company's ...

Dyne Therapeutics is advancing its investigational exon-skipping therapy zeleciment rostudirsen (z-rostudirsen), formerly known as DYNE-251, toward regulatory approval after trial data showed early ...

Columnist Betty Vertin says her three sons with DMD may not have a big social life, but they have a big family that makes up for it.

Dyne Therapeutics' Z-basivarsen led to gains in motor function and cognition for myotonic dystrophy type 1, and is entering a ...

The MDA's annual conference highlighted recent breakthroughs and the outlook for future care in the neuromuscular disease community.

Columnist Shalom Lim is using his lived experience with Duchenne muscular dystrophy to consult with organizations about ...

Columnist Robin Stemple knows that his physical challenges aren't his fault, but that doesn't make it any easier to avoid ...

Capricor has reapplied for FDA approval of deramiocel, its therapy for DMD-related heart disease, and expects a decision by August 22.

In 2018, when Nell Choi was 9 years old, she began experiencing symptoms that led to hospitalization and a diagnosis of neuromyelitis optica spectrum disorder (NMOSD), type positive. A month later, ...