Blocking the protein receptor IGF2R could promote muscle regeneration and improve muscle strength in people with Duchenne muscular…
Articles by Marisa Wexler, MS
Following its failure to increase dystrophin levels of patients in an early-stage clinical trial, Wave Life Sciences has discontinued…
A new technique that compares RNA production between an individual’s two gene copies may help find genetic alterations that lead…
Owing to a serious adverse event in a patient, the U.S. Food and Drug Administration (FDA) has put another clinical…
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…