M. Iammarino

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 · 392 citations · Full text

Pharmacy, Pediatrics, Pharmacotherapy +17

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

Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes

Megan A. Waldrop, Cassandra Karingada, Mike A. Storey et al. · PEDIATRICS · 2020 · 134 citations

Medicine, Genetics, Pediatrics +16

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