AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort - Concepedia

Concepedia

Publication | Open Access

AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort

DOI Full Paper Access

175

Citations

16

References

2019

Year

Samiah Al-Zaidy, Stephen J. Kolb, Linda Lowes, Lindsay N. Alfano, Richard Shell, Kathleen R. Church, Sukumar Nagendran, Douglas M. Sproule, Douglas E. Feltner, Courtney Wells,

Journal of Neuromuscular Diseases

Abstract

In this study, AVXS-101 increased the probability of survival, rapidly improved motor function, and enabled motor milestone achievement in SMA1 infants.

References

	Year	Citations

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