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Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy |
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| Authors: | Samiah Al‐Zaidy A. Simon Pickard Kavitha Kotha Lindsay N. Alfano Linda Lowes Grace Paul Kathleen Church Kelly Lehman Douglas M. Sproule Omar Dabbous Benit Maru Katherine Berry W. David Arnold John T. Kissel Jerry R. Mendell Richard Shell |
| Abstract: | |
| Keywords: | AVXS‐101 gene therapy gene replacement health outcomes quality of life SMA1 spinal muscular atrophy |