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 ...

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.

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 ...

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 ...

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 ...

Note: This story was updated Jan. 17, 2025, to clarify the company’s comments on gene therapies, which generally deliver a smaller form of dystrophin. This designation aims to encourage the ...