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SMA THERAPIES I: P.178AVXS-101 phase 1 gene therapy clinical trial in spinal muscular atrophy type 1: improvement in respiratory and bulbar function reduces frequency and duration of hospitalizations compared to natural history.

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Bibliographic Details
Title: SMA THERAPIES I: P.178AVXS-101 phase 1 gene therapy clinical trial in spinal muscular atrophy type 1: improvement in respiratory and bulbar function reduces frequency and duration of hospitalizations compared to natural history.
Authors: Shell, R.1, Al-Zaidy, S.1, Arnold, W.2, Rodino-Klapac, L.1, Prior, T.2, Kotha, K.1, Paul, G.1, Lowes, L.1, Alfano, L.1, Berry, K.1, Church, K.1, Kissel, J.2, Nagendran, S.3, Ogrinc, F.3, Sproule, D.3, Wells, C.3, Meyer, K.1, Likhite, S.1, Kaspar, B.3, Mendell, J.1
Source: Neuromuscular Disorders. Oct2018:Supplement 2, Vol. 28, pS82-S82. 1p.
Database: Academic Search Ultimate
Description
ISSN:09608966
DOI:10.1016/j.nmd.2018.06.206