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

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

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

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

Columnist Betty Vertin is grateful to have the help of a hired caregiver, but it's also been a difficult adjustment, she says.

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

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

Gene therapy designed for muscle cells. SRP-9003 is designed to deliver a healthy copy of the SGCB gene to muscle cells, to enable beta-sarcoglycan protein production.. In order to deliver its genetic ...

During a recent facioscapulohumeral muscular dystrophy (FSHD) wellness group meeting via Zoom, we each shared how we were feeling about our disease journey. One participant said, “I feel like I’m ...

As a person living with DMD, these discussions struck a chord with me, because these types of issues profoundly affect my daily life.Healthcare interactions with patients with Duchenne often involve ...

IPS Heart is also developing GIVI-MPC, another stem cell therapy designed to produce new skeletal muscle in people with DMD and BMD. This therapy has been designated as an orphan drug in the European ...