"The Times They Are a-Changin'." In reply to El-Zaidy et al.: AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort.
Servais, Laurent
2019 • In Journal of Neuromuscular Diseases, 6 (3), p. 319-320
Servais, Laurent ; Université de Liège - ULiège > Département des sciences cliniques > Département des sciences cliniques
Language :
English
Title :
"The Times They Are a-Changin'." In reply to El-Zaidy et al.: AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort.
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Al-Zaidy SA, Kolb SJ, Lowes L, Alfano LN, Shell R, Church KR, Nagendran S, Sproule DM, Feltner DE, Wells C, Ogrinc F, Menier M, L'Italien J, David Arnold W, Kissel JT, Kaspar BK, Mendell JR. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: comparative study with a prospective natural history cohort. Journal of Neuromuscular Diseases. In press.
