Katherine Berry

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

Longitudinal effect of eteplirsen versus historical control on ambulation in <scp>D</scp> uchenne muscular dystrophy

Jerry R. Mendell, Nathalie Goemans, Linda Lowes et al. · Annals of Neurology · 2015 · 504 citations · Full text

Medicine, Pharmacotherapy, Rehabilitation Medicine +13

A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy

Jerry R. Mendell, Zarife Sahenk, Vinod Malik et al. · Molecular Therapy · 2014 · 232 citations · Full text

Medicine, Genetics, Molecular Medicine +14

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

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