Kathleen Church

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 · 2.3K citations · Full text

Medicine, Genetics, Neurology +16

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

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 · 219 citations · Full text

Medicine, Genetics, Pediatrics +13

Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy

Linda Lowes, Lindsay N. Alfano, W. David Arnold et al. · Pediatric Neurology · 2019 · 181 citations · Full text

Medicine, Neurology, Pediatrics +15

Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes

Jerry R. Mendell, Zarife Sahenk, Samiah Al-Zaidy et al. · Molecular Therapy · 2017 · 105 citations · Full text

Medicine, Orthopedic Surgery, Rehabilitation Medicine +17