P.351Gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): long-term follow-up from the onasemnogene abeparvovec phase 1/2a clinical trial

Autor:	M. Iammarino, Linda Lowes, Richard Shell, N. Miller, J. Mendell, Francis G. Ogrinc, James L’Italien, Elaine Kernbauer, Kelly J. Lehman, M. McColly, Douglas E. Feltner, Douglas M. Sproule, H. Ouyang, Lindsay N. Alfano, S. Shah, K. Church, Samiah Al-Zaidy
Rok vydání:	2019
Předmět:	Clinical trial medicine.medical_specialty Neurology Long term follow up business.industry Pediatrics Perinatology and Child Health medicine Neurology (clinical) Spinal muscular atrophy business medicine.disease Genetics (clinical) Surgery
Zdroj:	Neuromuscular Disorders. 29:S184
ISSN:	0960-8966
DOI:	10.1016/j.nmd.2019.06.513
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_________::a07433721624af2e638ec352a9674222 https://doi.org/10.1016/j.nmd.2019.06.513 Zobrazit plný text záznamu Full Text from ScienceDirect