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

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.

The investigational therapy DYNE-101 worked as expected and led to functional improvements for people with myotonic dystrophy type 1 (DM1) in a clinical trial, according to an update from developer ...

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 ...