Scientists now have evidence to explain why treatment with the corticosteroid prednisone — but not the newer steroid Agamree (vamorolone) — drives bone loss in young boys with Duchenne muscular dystrophy (DMD) and may slow their growth. Using data from a clinical trial, the researchers found that…

I have lived with limb-girdle muscular dystrophy (LGMD) type 2E/R4 for more than 40 years. Initially, apart from stretching and visiting a neuromuscular clinic twice a year, I didn’t think much about managing my symptom progression. So much has changed. In January 2024, I was forced to put my…

With seven children — Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and Callie, 4; three of whom have Duchenne muscular dystrophy (DMD) — I have entered the zone of a veteran parent and caregiver. Max, Rowen, and Charlie were diagnosed with DMD in…

On April 25, 2016, my son Billy stood at a podium in a hotel ballroom outside Washington, D.C., and told a room full of advisers from the U.S. Food and Drug Administration (FDA) something no 15-year-old should have to say out loud. “FDA, please don’t let me die early,” he…

The U.S. Food and Drug Administration (FDA) has granted both fast track and orphan drug designations to Scholar Rock‘s apitegromab as a potential treatment for facioscapulohumeral muscular dystrophy (FSHD), a disease type that characteristically affects muscles of the face and upper body. The experimental…