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 ...

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

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.

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

PGN-EDODM1 has been tested in a Phase 1 study called FREEDOM-DM1 (NCT06204809) and results from the participants who took the 15 mg/kg dose are expected this year.. A Phase 2 study called FREEDOM2-DM1 ...

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 ...

DMD is caused by mutations in the gene DMD, which provides instructions to make dystrophin, a protein that helps maintain muscle health.Due to these mutations, people with DMD produce virtually no ...

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 ...