OUR APPROACH

References

1. Novartis AG (2021, October 26). Q3 2021 Results. https://www.novartis.com/investors/financial-data/quarterly-results. Accessed January 6, 2022
2. Weiβ, C.; Ziegler, A.; Becker, L., et al. (2021) Real-World Data for Onasemnogen Abeparvovec (Zolgensma) in Spinal Muscular Atrophy. Neuropediatrics 2021;52:S1-S53
3. FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality. May 24, 2019. https://www.fda.qovinews-events/press-a nnouncements/fda-approves-innovative-qene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease. Accessed January 6, 2022
4. Gessler D. J., Li D., Xu H., Su Q., Sanmiguel J., Tuncer S., et al. (2017). Redirecting N-acetylaspartate metabolism in the central nervous system normalizes myelination and rescues Canavan disease. JCI Insight 2:e90807. 10.1172/jci.insight.90807
5. New Zolgensma data demonstrate age-appropriate development when used early, real-world benefit in older children and durability 5+ years post-treatment. March 15, 2021. https://www.novartis.cominews/media-releases/new-zolqensma-data-demonstrate-aqe-appropriate-development-when-used-early-real-world-benefit-older-children-and-durability-5-years-post-treatment. Accessed January 6, 2022