Risdiplam: First Approval
Author:
Publisher
Springer Science and Business Media LLC
Subject
Pharmacology (medical)
Link
https://link.springer.com/content/pdf/10.1007/s40265-020-01410-z.pdf
Reference23 articles.
1. Ratni H, Ebeling M, Baird J, et al. Discovery of risdiplam, a selective survival of motor neuron-2 ( SMN2) gene splicing modifier for the treatment of spinal muscular atrophy (SMA). J Med Chem. 2018;61(15):6501–17.
2. Poirier A, Weetall M, Heinig K, et al. Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. Pharmacol Res Perspect. 2018;6(6):e00447.
3. 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.
4. Wang J, Schultz PG, Johnson KA. Mechanistic studies of a small-molecule modulator of SMN2 splicing. Proc Natl Acad Sci USA. 2018;115(20):E4604–E46124612.
5. Food and Drug Administration (FDA). FDA approves oral treatment for spinal muscular atrophy [media release]. 7 Aug 2020. https://www.fda.gov/news-events/.
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