"Nearly 25 years ago, a Mississippian visited my office and told me his son had been diagnosed with DMD... It has been among my greatest joys in Congress to help combat DMD and other muscular dystrophies ... I will continue to bring awareness to DMD.
) Positive Topline Results from Phase 1/2 DELIVER Trial of Zeleciment Rostudirsen (DYNE-251) in DMD to Support Potential ...Ravi, living with DMD ... Positive results from Registrational Expansion Cohort of DELIVER trial of z-rostudirsen in exon 51 DMD.
Elevidys is the only FDA-approved gene therapy for Duchenne’s Muscular Dystrophy (DMD), an incurable rare genetic disease that causes irreversible muscle loss at a fatal rate.
Having shared the data with members of our scientific steering committee, all KOLs in the DMD field, we are very encouraged by their response ... AGAMREE for the treatment of DMD is approved in the U.S.
53 LR Five-year data of AGAMREE® (vamorolone) in patients with DMD show improved safety profile with comparable effectiveness to standard of care corticosteroids ... AGAMREE for the treatment of DMD is approved in the U.S.
The biopharmaceutical company revealed that its Phase 3 ESSENCE study, evaluating the efficacy of its Duchenne muscular dystrophy (DMD) therapies Amondys 45 (casimersen) and Vyondys 53 (golodirsen), failed to meet its primary endpoint.