Statistics
Publications
347
Citations
52.2K
Authors
19
Location
Bannockburn, United States
347
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
Guy A. Van Hazel, Volker Heinemann, Navesh Sharma et al. · Journal of Clinical Oncology · 2016 · 348 citations
Ralf Ulrich Trappe, Daan Dierickx, Heiner Zimmermann et al. · Journal of Clinical Oncology · 2017 · 224 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 · 219 citations · Full text
Medicine, Genetics, Pediatrics +13
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