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Pediatric Neurology|December 3, 2014
Gene therapy for muscular dystrophy: moving the field forwardSamiah Al-Zaidy, Louise Rodino-Klapac, Jerry R MendellDevelopmental Medicine and Child Neurology|April 10, 2019
ACTIVE (Ability Captured Through Interactive Video Evaluation) workspace volume video game to quantify meaningful change in spinal muscular atrophyLindsay N Alfano, Natalie F Miller, Megan A Iammarino, et al.Journal of Child Neurology|October 9, 2020
Health-Related Quality of Life and Emotional Distress Among Mothers of Sons With Muscular Dystrophy as Compared to Sex- and Age Group-Matched ControlsJamie L Jackson, Christina X Korth, Carine E Leslie, 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.Molecular Therapy : the Journal of the American Society of Gene Therapy|March 11, 2017
Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional OutcomesJerry R Mendell, Zarife Sahenk, Samiah Al-Zaidy, 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.Molecular Therapy : the Journal of the American Society of Gene Therapy|October 18, 2014
A phase 1/2a follistatin gene therapy trial for becker muscular dystrophyJerry R Mendell, Zarife Sahenk, Vinod Malik, 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 1