News

Gene Variations May Benefit Those with Duchenne Muscular Dystrophy

Gene variations that decrease the production of a certain protein appear to reduce muscle strength in patients with Duchenne muscular dystrophy (DMD) but improve their disease progression, according to a study. The research, “Evidence For ACTN3 As A Genetic Modifier Of Duchenne Muscular Dystrophy,” was published in the journal…

Part B of MoveDMD Trial Shows Mixed Efficacy Results for CAT-1004 as Duchenne Treatment

Catabasis Pharmaceuticals reported mixed topline data from Part B of the MoveDMD trial evaluating the safety and efficacy of CAT-1004 (edasalonexent) in treating Duchenne muscular dystrophy (DMD) patients. MoveDMD is a three-part, multisite Phase 1/2 trial (NCT02439216) evaluating the efficacy, safety, and the pharmacodynamics (PD) and pharmacokinetics (PK),…