From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1

Published 2019 View Full Article

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Title
From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1
Authors
Keywords
AVXS-101, Zolgensma, Onasemnogene abeparvovec, Gene replacement, Gene therapy, Practical considerations, SMA1, Spinal muscular atrophy
Journal
PEDIATRIC NEUROLOGY
Volume -, Issue -, Pages -
Publisher
Elsevier BV
Online
2019-06-13
DOI
10.1016/j.pediatrneurol.2019.06.007

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