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

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

Brenda Song, MD, is a neurologist who directs the UMass Duchenne Muscular Dystrophy Program, and she is a pioneer in organizing multidisciplinary care for Duchenne populations. She explains how ...

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 ...

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.

Health Canada is granting priority review to givinostat, sold as Duvyzat in the U.S., as an oral treatment for Duchenne ...

Histone deacetylase inhibitors are a treatment approach designed to work by blocking an enzyme that turns off gene activity. This may ultimately help by slowing muscle loss and disease progression.

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 ...

Bridgebio Pharma has submitted an application asking the U.S. Food and Drug Administration (FDA) to approve its experimental oral therapy, BBP-418, to treat limb-girdle muscular dystrophy type 2i ...

Capricor Therapeutics will resubmit its application for U.S. Food and Drug Administration (FDA) approval of its cell therapy deramiocel to treat Duchenne muscular dystrophy (DMD)-related heart disease ...