Owing to a serious adverse event in a patient, the U.S. Food and Drug Administration (FDA) has put another clinical…
Articles by Marisa Wexler, MS
A novel one-time gene therapy that leads to the production of a dystrophin-related protein called utrophin prevents muscle…
Increasing the amount of a protein called sarcospan may help to protect against heart failure in children with Duchenne muscular…
Catabasis Pharmaceuticals announced new preclinical data showing that, in a mouse model of Duchenne muscular dystrophy (DMD),…
Biotechnology company Vertex has acquired Exonics Therapeutics, which focuses on using gene-editing technologies to treat Duchenne muscular dystrophy…
People with facioscapulohumeral muscular dystrophy (FSHD) are “very enthusiastic” about participating in clinical trials and studies, according to researchers,…
Very young boys with Duchenne muscular dystrophy (DMD) have measurable differences in motor skills, which could have implications for…
RG6206 (BMS-986089), a blocker of muscle growth-inhibitor myostatin, is safe to use in boys with Duchenne muscular dystrophy (DMD), results…