Itvisma expands the frontiers of gene replacement therapy for spinal muscular atrophy
-
-
REFERENCES
[1] Lefebvre S., Bürglen L., Reboullet S., et al. (1995). Identification and characterization of a spinal muscular atrophy-determining gene. Cell 80:155−165. DOI:10.1016/0092-8674(95)90460-3 [2] Wang D., Tai P.W.L. and Gao G. (2019). Adeno-associated virus vector as a platform for gene therapy delivery. Nat. Rev. Drug Discov. 18:358−378. DOI:10.1038/s41573-019-0012-9 [3] Proud C.M., Vũ D.C., Wilmshurst J.M., et al. (2025). Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: A phase 3 randomized controlled trial. Nat. Med. 32:481−487. DOI:10.1038/s41591-025-04103-w [4] Finkel R.S., Mercuri E., Darras B.T., et al. (2017). Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N. Engl. J. Med. 377:1723−1732. DOI:10.1056/NEJMoa1702752 [5] Mendell J.R., Al-Zaidy S., Shell R., et al. (2017). Single-dose gene-replacement therapy for spinal muscular atrophy. N. Engl. J. Med. 377:1713−1722. DOI:10.1056/NEJMoa1706198 -
ABOUT THIS ARTICLE
Cite this article:
The Innovation Medicine News Team (2026). Itvisma expands the frontiers of gene replacement therapy for spinal muscular atrophy. The Innovation Medicine 4:100243. https://doi.org/10.59717/j.xinn-med.2026.100243
Welcome!
To request copyright permission to republish or share portions of our works, please visit Copyright Clearance Center's (CCC) Marketplace website at marketplace.copyright.com.
Export File
Citation
The Innovation Medicine News Team (2026). Itvisma expands the frontiers of gene replacement therapy for spinal muscular atrophy. The Innovation Medicine 4:100243. https://doi.org/10.59717/j.xinn-med.2026.100243
Format
Content
DownLoad:
-
Figure 1.
Expanding gene replacement therapy across the SMA treatment spectrum.
