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Gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): Long-term follow-up from the onasemnogene abeparvovec phase 1/2A clinical trial

Document Type

Abstract

Author

Mendell, J.R.; Shell, R.; Lehman, K.J.; McColly, M.; Lowes, L.P.; Alfano, L.N.; Miller, N.F.; Iammarino, M.A.; Church, K.; Spector, S.A.; Ogrinc, F.G.; Ouyang, H.; Kernbauer, E.; Shah, S.; L’Italien, J.; Sproule, D.M.; Feltner, D.E.; Al-Zaidy, S.

Source

In Journal of the Neurological Sciences 15 October 2019 405 Supplement:271-271

Subject

WCN19-1340

Language

ISSN

0022-510X

Online Access

Full Text (ScienceDirect 종량제) Full Text (Clinical Key) Web of Science JCR 저널정보 Scopus Find it@PNU

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