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

Autor:	N.F. Miller, L.P. Lowes, K. Church, Samiah Al-Zaidy, Lindsay N. Alfano, J. Mendell, Elaine Kernbauer, S. Shah, James L’Italien, F.G. Ogrinc, Kelly J. Lehman, Richard Shell, S.A. Spector, Douglas M. Sproule, M. A. Iammarino, Douglas E. Feltner, Markus McColly, H. Ouyang
Rok vydání:	2019
Předmět:	Clinical trial medicine.medical_specialty Neurology business.industry Long term follow up Gene replacement therapy medicine Neurology (clinical) Spinal muscular atrophy medicine.disease business Surgery
Zdroj:	Journal of the Neurological Sciences. 405:271
ISSN:	0022-510X
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_________::fb13aa8e4a16839778c64130ce003663 https://doi.org/10.1016/j.jns.2019.10.1322 Zobrazit plný text záznamu Full Text from ScienceDirect