News

Another gene therapy death of a Duchenne patient has some parents and doctors criticizing the FDA's actions, even as other ...
Capricor Therapeutics has received an orphan drug designation for its drug aimed at potentially treating Becker Muscular Dystrophy. The biotechnology company said Tuesday that the Food and Drug ...
Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying ...
Organoids Reveal Similarities Between Myotonic Dystrophy Type 1 and Rett Syndrome June 29, 2022 — Using brain organoids, researchers discover mutational commonalities between muscular dystrophy ...
Muscular Dystrophy Association ... National Organization for Rare Disorders: “Kugelberg Welander Syndrome.” Spinal Muscular Atrophy UK: “Symptoms, Diagnosis, and Effects of 5q Spinal ...
We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...
Disclosing new interim data from its Phase 1/2 AFFINITY DUCHENNE trial, Regenxbio (NASDAQ:RGNX) announced Thursday that its ...
The campaign to commemorate the work of the Muscular Dystrophy Association was created in partnership with agency Yes&.