The FDA has granted rare pediatric disease designation to PBGENE-DMD, an experimental gene-editing therapy for Duchenne ...

A recent dream leaves columnist Robin Stemple questioning how his disability progression due to FSHD affects his family and ...

The U.S. Food and Drug Administration is due to review data on deramiocel, a cell therapy up for approval for heart disease ...

Caregiving means that columnist Betty Vertin sometimes misses out on activities with her children who don't have DMD. Finding balance is key.

A recent concert in Singapore was all about finding harmony through the talents that unite us in life, writes columnist.

For the second time, a person with DMD has died of acute liver failure after being treated with the one-time gene therapy Elevidys.

As her only son without DMD plays in a statewide all-star football game, columnist Betty Vertin reflects on his tie to his brothers.

It’s been a couple of weeks since my last column. I’ve missed writing them. I can never explain how therapeutic it is for me to share my life as a caregiver through writing. I have seven children: ...

ARM-201 is designed to block the production of DUX4, the protein that is abnormally produced in people with FSHD, a type of muscular dystrophy that primarily affects the muscles of the face, shoulders ...

DMD is caused by mutations in the DMD gene, which provides instructions for making dystrophin, a protein essential for maintaining muscle health. Elevidys delivers a gene that encodes a shortened, but ...

Decker, who has limb-girdle muscular dystrophy (LGMD) type 2D, was honored at the Muscular Dystrophy Association (MDA)’s annual meeting, recently held in Dallas and virtually. “It’s always nice to be ...

In a statement, the therapy’s developer, Sarepta Therapeutics, said it is continuing to gather and analyze information about this event, which has been reported to relevant health authorities.Elevidys ...