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

2020 ◽  
Author(s):  
R. Shell ◽  
J.W. Day ◽  
C.A. Chiriboga ◽  
T.O. Crawford ◽  
B.T. Darras ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Replacement Therapy ◽  
Muscular Atrophy ◽  
Gene Replacement ◽  
Phase 3 ◽  
Pivotal Phase ◽  
Spinal Muscular Atrophy Type ◽  
Gene Replacement Therapy

scholarly journals Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy

2018 ◽  
Vol 54 (2) ◽  
pp. 179-185 ◽  
Author(s):  
Samiah Al‐Zaidy ◽  
A. Simon Pickard ◽  
Kavitha Kotha ◽  
Lindsay N. Alfano ◽  
Linda Lowes ◽  
...  
Keyword(s):  
Health Outcomes ◽  
Spinal Muscular Atrophy ◽  
Replacement Therapy ◽  
Muscular Atrophy ◽  
Gene Replacement ◽  
Spinal Muscular Atrophy Type ◽  
Gene Replacement Therapy
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