P.066 AVXS-101 gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): long-term follow-up from the phase 1 clinical trial

Title
P.066 AVXS-101 gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): long-term follow-up from the phase 1 clinical trial
Authors
Keywords
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Journal
CANADIAN JOURNAL OF NEUROLOGICAL SCIENCES
Volume 46, Issue s1, Pages S32
Publisher
Cambridge University Press (CUP)
Online
2019-06-05
DOI
10.1017/cjn.2019.166

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