A Phase 3 clinical trial testing delpacibart etedesiran (del-desiran), an experimental treatment for myotonic dystrophy type 1 (DM1), failed to hit its main goal. The Phase 3 HARBOR trial (NCT06411288) enrolled nearly 160 people with DM1, ages 16 to 65. Participants received seven infusions of either del-desiran or…

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…