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…

September is Muscular Dystrophy Awareness Month, and advocates are launching campaigns to educate people about the different types of rare genetic muscle disorders that comprise muscular dystrophy (MD) — and to fundraise to fuel research. Still, the focus is on spotlighting those living with MD, Kelly Berger, community engagement…

The house is quiet this morning. A dog sleeps next to me on the couch as I contemplate the words I will use to write this column. The news is on, so there is some background noise, and I can faintly hear a video game playing in my son’s…

For the first time, a person with Duchenne muscular dystrophy (DMD) has been given an experimental gene-editing therapy, called PBGENE-DMD, that’s designed to treat the neurodegenerative disease by altering the patient’s own genetic code. Precision Biosciences, the U.S. company developing PBGENE-DMD, announced that dosing had begun in the…