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

2019176 citationsJournal Articlebronze Open Access

Authors

Samiah Al-Zaidy · Nationwide Children's Hospital

Stephen J. Kolb · The Ohio State University Wexner Medical Center

Linda Lowes · Nationwide Children's Hospital

Lindsay N. Alfano · Nationwide Children's Hospital

Richard Shell · The Ohio State University Wexner Medical Center

Kathleen R. Church · Nationwide Children's Hospital

Sukumar Nagendran

Abstract

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

Topics & Keywords

Neurogenetic and Muscular Disorders Research Congenital Anomalies and Fetal Surgery RNA modifications and cancer

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: Journal of Neuromuscular Diseases

Volume 6, Issue 3, pp. 307-317

DOI: 10.3233/jnd-190403

Field-Weighted Citation Impact: 15.40