AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort-Reference-Cited by-同舟云学术

AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort

Published:2019-09-03 Issue:3 Volume:6 Page:307-317
ISSN:2214-3599
Container-title:Journal of Neuromuscular Diseases
language:
Short-container-title:JND

Author:

Al-Zaidy Samiah A.¹²,Kolb Stephen J.³⁴,Lowes Linda¹²,Alfano Lindsay N.¹²,Shell Richard²,Church Kathleen R.¹,Nagendran Sukumar⁵,Sproule Douglas M.⁵,Feltner Douglas E.⁵,Wells Courtney⁵,Ogrinc Francis⁵,Menier Melissa⁵,L’Italien James⁵,Arnold W. David³,Kissel John T.²,Kaspar Brian K.⁵,Mendell Jerry R.¹²³

Affiliation:

1. Center for Gene Therapy Research Institute, Nationwide Children’s Hospital, Columbus, OH, USA

2. Department of Pediatrics, The Ohio State University Wexner Medical Center, Columbus, OH, USA

3. Department of Neurology, The Ohio State University Wexner Medical Center, Columbus, OH, USA

4. Department of Biological Chemistry and Pharmacology, The Ohio State University Wexner Medical Center, Columbus, OH, USA

5. AveXis, Inc., Bannockburn, IL, USA

Publisher

IOS Press

Subject

Clinical Neurology,Neurology

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3. Observational study of spinal muscular atrophy type I and implications for clinical trials;Finkel;Neurology,2014

4. Natural history of infantile-onset spinal muscular atrophy;Kolb;Ann Neurol,2017

5. Developmental milestones in type I spinal muscular atrophy;De Sanctis;Neuromuscul Disord,2016

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