The FDA is reviewing full approval bids for the Duchenne therapies Amondys 45 and Vyondys 53, with decisions expected by ...

A CITGO golf fundraiser in Louisiana raised a record $756,800 for MDA to support people living with neuromuscular diseases ...

Being part of the PPMD conference's Adult Advisory Committee has helped columnist Patrick Moeschen bond with those who share ...

On July 29, Capricor Therapeutics will meet with an FDA advisory committee regarding its second attempt to win deramiocel's approval for DMD.

MDA criticized a DOJ memorandum on disability care, saying it threatens in-home and community-based services for people with ...

Columnist Betty Vertin has been worried about weight gain and her stress levels, but the results of her annual physical were good news.

Rob Stemple is a lifelong advocate for people with disabilities. He was diagnosed with FSHD in 1971 at age 14. Rob struggled with its’ affects for over 50 years. He lost his eyesight in a devastating ...

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations to GEn1E Lifesciences‘ GEn-1123 as a potential treatment for Duchenne muscular ...

Exon skipping is a treatment approach for people whose Duchenne muscular dystrophy is due to certain mutations in its causative gene. Exon skipping works like a molecular patch, so that the DMD gene ...

People with Duchenne muscular dystrophy (DMD) require some level of caregiving throughout their whole lives, particularly as the disease progresses and patients become less mobile and more reliant on ...

A young man with Duchenne muscular dystrophy (DMD) who received the one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl) has died due to acute liver failure. In a statement, the ...

Muscular dystrophy entered my life in the fall of 1984. I was 11 years old and beginning my sixth year of school. Along with neighborhood friends, I would walk to and from my elementary school every ...