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Annals of Neurology|June 3, 2010
Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophyVinod Malik, Louise R Rodino-Klapac, Laurence Viollet, et al.Frontiers in Cell and Developmental Biology|July 27, 2023
Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophyJerry R Mendell, Perry B Shieh, Craig M McDonald, et al.Annals of Neurology|August 3, 2013
Eteplirsen for the treatment of Duchenne muscular dystrophyJerry R Mendell, Louise R Rodino-Klapac, Zarife Sahenk, et al.Neurology and Therapy|January 10, 2026
Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK TrialJerry R Mendell, Francesco Muntoni, Craig M McDonald, et al.Nature Medicine|August 11, 2014
Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and miceNicolas Wein, Adeline Vulin, Maria S Falzarano, et al.Nature Medicine|October 9, 2024
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trialJerry R Mendell, Francesco Muntoni, Craig M McDonald, et al.The New England Journal of Medicine|November 2, 2017
Single-Dose Gene-Replacement Therapy for Spinal Muscular AtrophyJerry R Mendell, Samiah Al-Zaidy, Richard Shell, et al.Pageof 7