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JAMA Neurology|May 17, 2021
Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular AtrophyJerry R Mendell, Samiah A Al-Zaidy, Kelly J Lehman, et al.Pediatric Neurology|July 16, 2021
Validity and Reliability of the Neuromuscular Gross Motor OutcomeLindsay N Alfano, Megan A Iammarino, Natalie F Reash, et al.Pediatric Pulmonology|December 15, 2018
Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapySamiah Al-Zaidy, A Simon Pickard, Kavitha Kotha, et al.Pediatric Neurology|July 7, 2019
Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement TherapyLinda P Lowes, Lindsay N Alfano, W David Arnold, et al.Muscle & Nerve|January 2, 2025
Transitioning From Nusinersen to Risdiplam for Spinal Muscular Atrophy in Clinical Practice: A Single-Center ExperienceCan Ebru Bekircan-Kurt, Sharmada Subramanian, Shannon Chagat, et al.Journal of Neuromuscular Diseases|August 6, 2019
AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History CohortSamiah A Al-Zaidy, Stephen J Kolb, Linda Lowes, et al.Muscle & Nerve|August 14, 2023
Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trialJerry R Mendell, Zarife Sahenk, Kelly J Lehman, et al.Neuromuscular Disorders : NMD|December 24, 2023
Continued safety and long-term effectiveness of onasemnogene abeparvovec in OhioMegan A Waldrop, Shannon Chagat, Michael Storey, et al.JAMA Neurology|June 16, 2020
Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled TrialJerry R Mendell, Zarife Sahenk, Kelly Lehman, 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 4