リスジプラム
Journal of Medicinal Chemistry (Washington, D.C.) 61 (15): 6501-6517. doi:10.1021/acs.jmedchem.8b00741. ISSN 00222623. LCCN a630-643. OCLC 39480771. PMID 30044619.
↑ Zanetta C, Nizzardo M, Simone C, Monguzzi E, Bresolin N, Comi GP, Corti S (January 2014). “Molecular therapeutic strategies for spinal muscular atrophies: current and future clinical trials”. Clinical Therapeutics (Philadelphia, Pennsylvania, United States of America: Elsevier) 36 (1): 128-140. doi:10.1016/j.clinthera.2013.11.006. PMID 24360800.
1 2 Baranello, G.; Servais, L.; Day, J.; Deconinck, N.; Mercuri, E.; Klein, A. et al. (1 October 2019). “P.
↑ Roche Group Media Relations Division (17 December 2018). "PRIME designation granted by European Medicines Agency for Roche's risdiplam for treatment of spinal muscular atrophy (SMA)" (Press release) (English). Basel, Switzerland: F. Hoffmann-La Roche Ltd. Group Communications Department (Roche Group Media Relations Division). pp. 1–5. 2018年8月18日時点のオリジナル (PDF)よりアーカイブ。2021年6月9日閲覧。
↑ Stoyanova-Beninska, Violeta (Committee Chair); Schwarzer-Daum, Brigitte (Committee Vice Chair) (26 February 2019). Public summary of opinion on orphan designation: Risdiplam for the treatment of spinal muscular atrophy (PDF). EMA Committee for Orphan Medicinal Products (COMP) (Report) (English). Amsterdam, The Netherlands: European Medicines Agency (EMA Committee for Orphan Medicinal Products).
脊髄性筋萎縮症(早産児および生後2か月未満の乳児に対する有効性および安全性は確立していない)