Four people with oculopharyngeal muscular dystrophy (OPMD), a disease type marked by swallowing difficulties, experienced long-lasting improvements in their…
Articles by Marisa Wexler, MS
Dosing has begun in a Phase 2 clinical trial testing the oral therapy SAT-3247 in boys with Duchenne muscular…
The U.S. Food and Drug Administration (FDA) has cleared Precision Biosciences to launch a clinical trial testing its gene-editing…
Three years after receiving the gene therapy Elevidys (delandistrogene moxeparvovec-rokl) in a clinical trial, boys with Duchenne muscular…
For people with Duchenne muscular dystrophy (DMD), 2026 promises to be a year of tremendous excitement, with several new…
Using a “pothole-filling” strategy that combines the precision of DNA-like strands with the versatility of small molecules, scientists have designed…
The Muscular Dystrophy Association (MDA) announced that registration is now open for its 2026 summer camp program. The…
Duchenne muscular dystrophy (DMD) causes dysfunction of muscle cells that help move blood through the circulatory system, a study…
Every newborn in the United States should now be screened for Duchenne muscular dystrophy (DMD), according to a recommendation from…
Dyne Therapeutics said it plans to ask the U.S. Food and Drug Administration (FDA) to approve DYNE-251, its exon-skipping…