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

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 ...

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 ...

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 European Comission has conditionally approved Duvyzat (givinostat) to be taken alongside corticosteroids for treating people who are 6 and older with Duchenne muscular dystrophy (DMD). “This ...

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