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

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

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

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.

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

With FSHD, the need to adapt seems never-ending, which is why columnist Robin Stemple and others feel like they're constantly ...

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