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.

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 ...

Several types of muscular dystrophy (MD) are rare, including my own form of it. This makes it difficult for me to find healthcare professionals who are experts in the field. I’ve had to travel by air ...

When I think about my son Charlie, I always remember my pregnancy with him. I’d taken a home test and knew I was pregnant, but when I called to make an appointment with my doctor to confirm the good ...

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 ...