| Title: |
AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort |
| Authors: |
Al-Zaidy, Samiah A.; Kolb, Stephen J.; Lowes, Linda; Alfano, Lindsay N.; Shell, Richard; Church, Kathleen R.; Nagendran, Sukumar; Sproule, Douglas M.; Feltner, Douglas E.; Wells, Courtney; Ogrinc, Francis; Menier, Melissa; L’Italien, James; Arnold, W. David; Kissel, John T.; Kaspar, Brian K.; Mendell, Jerry R. |
| Source: |
Journal of Neuromuscular Diseases ; volume 6, issue 3, page 307-317 ; ISSN 2214-3599 2214-3602 |
| Publisher Information: |
SAGE Publications |
| Publication Year: |
2019 |
| Document Type: |
article in journal/newspaper |
| Language: |
unknown |
| DOI: |
10.3233/jnd-190403 |
| DOI: |
10.3233/JND-190403 |
| Availability: |
https://doi.org/10.3233/jnd-190403; https://content.iospress.com/download?id=10.3233/JND-190403 |
| Rights: |
https://creativecommons.org/licenses/by-nc/4.0/ |
| Accession Number: |
edsbas.C358072B |
| Database: |
BASE |