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 ...

Last Thursday, I participated in a workshop that used experiential theater to examine healthcare conversations. With a sense of anticipation and hope, I stepped into the unknown, eager to gain ...

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