Top-line Phase 3 trial data show ambulatory boys with Duchenne continued with motor improvements two years after a single ...

Columnist Shalom Lim is working with Julia Cameron's popular book "The Artist's Way" as a route to develop his creative self, ...

Columnist Robin Stemple has to make yet another adjustment due to FSHD, after a fall left him feeling "like an upside-down ...

A pilot study in 19 LGMD patients and one with Becker MD found low-dose prednisone helped lower markers of muscle damage and ...

The FDA has granted fast track designation to DYNE-101, a therapy for myotonic dystrophy type 1 (DM1) now in testing in a ...

DYNE-101 eased myotonia and led to other functional improvements for people with myotonic dystrophy type 1 (DM1) in a ...

GIVI-MPC, a stem cell therapy for certain types of muscular dystrophy, has been granted orphan drug status for treating Becker MD.

As a parent and caregiver to three sons with DMD, columnist Betty Vertin's definition of what success looks like has changed over the years.

The U.S. Food and Drug Administration (FDA) has granted fast track designation to DYNE-101, a treatment candidate for myotonic dystrophy type 1 (DM1) — in which disease symptoms begin during adulthood ...

A gene-editing therapy designed to correct a defect in the DYSF gene — one that’s associated with a form of limb-girdle muscular dystrophy, or LGMD — was found to restore dysferlin protein levels and ...