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

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.

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

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

Columnist Patrick Moeschen was given an incorrect diagnosis of Becker muscular dystrophy, years before learning he actually ...

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

I live in rural Nebraska with my husband and our seven children. Three of them — Max, 19; Rowen, 15; and Charlie, 14 — live with Duchenne muscular dystrophy (DMD). People who meet me in person for the ...

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 ...

Many times over the years since the 1989 car crash that nearly killed me, I’ve been asked if I had any near-death or out-of-body experiences while I hung between life and death. I always try to ...