AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Event free survival and achievement of developmental milestones
2017 ◽
Vol 21
◽
pp. e13-e14
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Jerry R. Mendell
◽
Samiah Al-Zaidy
◽
Richard Shell
◽
W. David Arnold
◽
Louise Rodino-Klapac
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...