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

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

An accident and a malfunction render both of columnist Betty Vertin's accessible vans useless, leaving the family without transportation.

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

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 ...

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 ...

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

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 ...