Dyne Therapeutics Announces Upcoming Presentations on Initial Clinical Data From its ACHIEVE Trial in DM1 Patients and DELIVER Trial in DMD Patients at the 2024 Muscular Dystrophy Association Clinical & Scientific Conference tmcnet.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from tmcnet.com Daily Mail and Mail on Sunday newspapers.
Nearly half of patients treated with zilucoplan were considered responders on Myasthenia Gravis Activities of Daily Living (MG-ADL) at the first week, suggesting a rapid onset of efficacy.
- Phase 2 open-label CONNECT1-EDO51 study expected to be initiated in Canada in the first half of 2023 - - Potentially registration-directed, randomized, double-blind, placebo-controlled Phase 2 CONNECT2-EDO51
Ataluren preserved muscle burst activity across a range of ambulatory tests compared with placebo in boys with nonsense mutation Duchenne muscular dystrophy, according to a poster at the 2023 MDA Clinical & Scientific Conference.Seeking to evaluate the effects of ataluren on muscle burst activity in boys with nonsense mutation Duchenne muscular dystrophy (nmDMD), Craig McDonald, MD, professor
Delandistrogene moxeparvovec improved motor function and was well-tolerated 4 years after treatment in a small cohort of children with Duchenne muscular dystrophy, according to a poster at the 2023 MDA Clinical & Scientific Conference.“Delandistrogene moxeparvovec is an investigational gene transfer therapy developed to address the root cause of Duchenne muscular dystrophy through