Natalie F. Reash

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 · 280 citations · Full text

Medicine, Neurology, Pediatrics +17

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 dystrophy

Jerry R. Mendell, Perry B. Shieh, Craig M. McDonald et al. · Frontiers in Cell and Developmental Biology · 2023 · 81 citations · Full text

Medicine, Molecular Medicine, Neurology +13

Long‐term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial

Jerry R. Mendell, Zarife Sahenk, Kelly J. Lehman et al. · Muscle & Nerve · 2023 · 76 citations · Full text

Medicine, Molecular Medicine, Gene Therapy +13

Validation of the North Star Assessment for Limb-Girdle Type Muscular Dystrophies

M. James, Lindsay N. Alfano, Robert Muni‐Lofra et al. · Physical Therapy · 2022 · 30 citations · Full text

Medicine, Neurology, Rehabilitation Medicine +17

Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results

Jerry R. Mendell, Eric R. Pozsgai, Sarah Lewis et al. · Nature Medicine · 2024 · 27 citations · Full text

Life Sciences, Biotechnology, Molecular Biology +17