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.

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

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

Columnist Robin Stemple marks two anniversaries that forever altered his life. Despite the incredible challenges, he feels ...

Duvyzat's approvals were based on the Phase 3 EPIDYS trial, which included 179 boys, ages 6 and older, who were able to walk.

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

Patients are often the best experts on their disease, but what about emergency situations? Columnist Patrick Moeschen shares his thoughts.

The U.S. Food and Drug Administration (FDA) granted platform technology designation to the viral vector used in SRP-9003, an investigational gene therapy for limb-girdle muscular dystrophy type 2E ...

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 ...

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 ...