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. 2022 Jun 16;16:1865–1883. doi: 10.2147/DDDT.S214174

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© 2022 Hjartarson et al.

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution – Non Commercial (unported, v3.0) License (http://creativecommons.org/licenses/by-nc/3.0/). By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms (https://www.dovepress.com/terms.php).

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Simplified illustration of the mechanism of action of the three disease modifying therapies for SMA: Onasemnogene abeparvovec, nusinersen and risdiplam.

Notes: Adapted from Schorling DC, Pechmann A, Kirschner J. Advances in treatment of spinal muscular atrophy - new phenotypes, new challenges, new implications for care. J Neuromuscul Dis. 2020;7(1):1–13. © 2020 – IOS Press and the authors. All rights reservedThis article is published online with Open Access and distributed under the terms of the Creative Commons Attribution Non-Commercial License (CC BY-NC 4.0).104

Abbreviations: SMN∆7, SMN protein lacking exon 7.