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

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 ...

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 ...

After taking a day for reflection on her sons' lives with DMD, columnist Betty Vertin says she feels more on top of their care.