Key milestones have been met in a clinical trial testing a one-time treatment that aims to turn off the faulty gene in FSHD, ...

Columnist Shalom Lim is grateful that his profile on the Purple Parade website highlights the importance of his disability ...

This year’s Be Their Muscle campaign has raised more than $641,000 to support the MDA Summer Camp and help advance research ...

After decades of advocacy work, columnist Patrick Moeschen is glad to see MD patients treated as experts given their lived ...

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

I have heard that said hundreds of times over the past 25 years, and I hated hearing it. I always had a response ready: “Sure, but my heart is fuller,” I would say. However, I’m currently in a season ...

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 ...

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

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 ...

Myostatin or growth differentiation factor 8 (GDF8) is a naturally occurring protein in muscles. It belongs to a family of proteins called transforming growth factor-beta or TGFβ. Myostatin, produced ...

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.