Onasemnogene Abeparvovec-xioi Gene-Replacement Therapy for Spinal Muscular Atrophy Type 1: Pulmonary and Ventilatory Findings from the Pivotal Phase 3 US Study (STR1VE)

Authors :: Thomas A. Macek
Edward C. Smith
John W. Day
Perry B. Shieh
S.T. Iannaconne
H. Ouyang
Anne M. Connolly
Richard Shell
Craig M. Zaidman
J. Mendell
Douglas M. Sproule
Thomas O. Crawford
Sitra Tauscher-Wisniewski
Claudia A. Chiriboga
Meredith Schultz
L.D.M. Peña
Richard S. Finkel
Basil T. Darras
Francis G. Ogrinc
P. Thomasma
Jennifer M. Kwon
Douglas E. Feltner
Nancy L. Kuntz
Elaine Kernbauer
Source :: C96. BEST OF PEDIATRICS.
Publication Year :: 2020
Publisher :: American Thoracic Society, 2020.

Subjects :: medicine.medical_specialty
business.industry
Internal medicine
Gene replacement therapy
medicine
Cardiology
Spinal muscular atrophy
medicine.disease
business

Database :: OpenAIRE
Journal :: C96. BEST OF PEDIATRICS
Accession number :: edsair.doi...........2e156b19a2b6acef12e6bd85c203bd32
Full Text :: https://doi.org/10.1164/ajrccm-conference.2020.201.1_meetingabstracts.a5974

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