Richard Shell

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Jerry R. Mendell, Samiah Al-Zaidy, Richard Shell et al. · New England Journal of Medicine · 2017 · 2.2K citations · Full text

Natural history of infantile‐onset spinal muscular atrophy

Stephen J. Kolb, Christopher S. Coffey, Jon Yankey et al. · Annals of Neurology · 2017 · 392 citations · Full text

Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy

Jerry R. Mendell, Zarife Sahenk, Kelly J. Lehman et al. · JAMA Neurology · 2020 · 360 citations · Full text

Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy

Jerry R. Mendell, Samiah Al-Zaidy, Kelly J. Lehman et al. · JAMA Neurology · 2021 · 262 citations · Full text

Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy

S. Al-Zaidy, A. Simon Pickard, Kavitha Kotha et al. · Pediatric Pulmonology · 2018 · 213 citations · Full text