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

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

The U.S. Food and Drug Administration (FDA) granted platform technology designation to the viral vector used in SRP-9003, an ...

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

Columnist Betty Vertin acknowledges the mixed feelings of fear and pride as her youngest son with DMD begins with high school band.

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ISX9-CPC, IPS Heart ’s experimental stem cell ...

Building empathy and having clear communication are key to improving patient outcomes in DMD, writes columnist.

Pepgen will stop development of its Duchenne MD exon-skipping therapy PGN-ED051 after increases in dystrophin levels proved ...