リスジプラム
参考資料

1 2  “Evrysdi”. Therapeutic Goods Administration (TGA) (11 June 2021). 6 September 2021閲覧。
1 2  “AusPAR: Risdiplam”. Therapeutic Goods Administration (TGA) (13 September 2021). 13 September 2021閲覧。
1 2 3 4 5 6 7 8  Food and Drugs Administration (FDA) (18 August 2020). “Evrysdi- risdiplam powder, for solution”. DailyMed.  MedLine by the National Library of Medicine (NLM) of the United States National Institutes of Health (NIH). 24 September 2020閲覧。
1 2 3 4 5 6 7 8 9 10 11 12 13 14  O'Keefe, Lindsey (7 August 2020). "FDA Approves Oral Treatment for Spinal Muscular Atrophy" (Press release). Silver Spring, Maryland, United States of America: United States Food and Drug Administration (FDA). FDA Newsroom Department. 2020年8月11日時点のオリジナルよりアーカイブ。2020年8月7日閲覧。  この記述には、アメリカ合衆国内でパブリックドメインとなっている記述を含む。
↑  “KEGG DRUG: リスジプラム”. www.genome.jp. 2021年10月18日閲覧。
1 2 3 4 5 6  Food and Drugs Administration (FDA) (7 August 2020). “Evrysdi (Risdiplam) for Spinal Muscular Atrophy”. SMA News Today (Pensacola, Florida, United States: BioNews Services (BioNews Services, LLC.)).  オリジナルの21 January 2021時点におけるアーカイブ。. https://web.archive.org/web/20210127163559/https://smanewstoday.com/evrysdi-risdiplam 9 June 2021閲覧。
1 2  Zhao X, Feng Z, Ling KK, Mollin A, Sheedy J, Yeh S, Petruska J, Narasimhan J, Dakka A, Welch EM, Karp G, Chen KS, Metzger F, Ratni H, Lotti F, Tisdale S, Naryshkin NA, Pellizzoni L, Paushkin S, Ko CP, Weetall M (May 2016). “Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy”. Human Molecular Genetics (Oxford, United Kingdom of Great Britain: Oxford University Press (OUP)) 25 (10):  1885-1899. doi:10.1093/hmg/ddw062. PMC 5062580. PMID 26931466. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5062580/.
1 2 3 4 5  "FDA Approves Genentech's Evrysdi (risdiplam) for Treatment of Spinal Muscular Atrophy (SMA) in Adults and Children 2 Months and Older". Genentech (Press release) (English). San Francisco, California, United States of America: Genentech, Inc. (Roche Group). Genentech Global Product Development Division. 7 August 2020. 2020年8月18日時点のオリジナルよりアーカイブ。2020年8月7日閲覧。
1 2  “2021年06月23日｜エブリスディ、脊髄性筋萎縮症に対し承認を取得｜ニュースリリース｜中外製薬”. 中外製薬企業情報サイト. 2021年10月18日閲覧。
1 2  “エブリスディドライシロップ60mg 添付文書”. www.info.pmda.go.jp. 2021年10月18日閲覧。
↑  佐橋健太郎、勝野雅央「脊髄性筋萎縮症に対する神経スプライシング制御治療」『神経治療』第37巻第3号、2020年、247-250頁、doi:10.15082/jsnt.37.3_247。
↑  Ramdas S, Servais L (February 2020). “New treatments in spinal muscular atrophy: an overview of currently available data”. Expert Opinion on Pharmacotherapy (London, England, United Kingdom of Great Britain: Taylor & Francis (Informa UK Ltd)) 21 (3):  307-315. doi:10.1080/14656566.2019.1704732. OCLC 57378019. PMID 31973611.
↑  “RG7916”.   BioNews Services (BioNews Services, LLC.) (8 November 2016). 24 April 2020時点のオリジナルよりアーカイブ。9 June 2021閲覧。
1 2  Baranello G, Darras BT, Day JW, Deconinck N, Klein A, Masson R, Mercuri E, Rose K, El-Khairi M, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Seabrook T, Fontoura P, Servais L (March 2021). “Risdiplam in Type 1 Spinal Muscular Atrophy”. The New England Journal of Medicine (Boston, Massachusetts, United States of America: NEJM Group (Massachusetts Medical Society)) 384 (10):  915-923. doi:10.1056/NEJMoa2009965. ISSN 00284793. LCCN 20-20456. OCLC 231027780. PMID 33626251.
↑  Ratni H, Ebeling M, Baird J, Bendels S, Bylund J, Chen KS, Denk N, Feng Z, Green L, Guerard M, Jablonski P, Jacobsen B, Khwaja O, Kletzl H, Ko CP, Kustermann S, Marquet A, Metzger F, Mueller B, Naryshkin NA, Paushkin SV, Pinard E, Poirier A, Reutlinger M, Weetall M, Zeller A, Zhao X, Mueller L (August 2018). “Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)”. 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.353FIREFISH Part 1: 16-month safety and exploratory outcomes of risdiplam (RG7916) treatment in infants with type 1 spinal muscular atrophy”. Neuromuscular Disorders (London, United Kingdom of Great Britain: World Muscle Society (WMS)/Elsevier Inc.) 29 (Supplement 1):  S184. doi:10.1016/j.nmd.2019.06.515. ISSN 0960-8966. OCLC 24318845.
1 2  “Update from SUNFISH Part 1: Safety, Tolerability and PK/PD from the Dose-Finding Study, Including Exploratory Efficacy Data in Patients with Type 2 or 3 Spinal Muscular Atrophy (SMA) Treated with Risdiplam (RG7916) (S25.007)”. Neurology (Minneapolis, Minnesota, United States of America: American Academy of Neurology/Wolters Kluwer) 92 (15 (Supplement)). (16 April 2019). ISSN 00283878. LCCN 55-43902. OCLC 960771045. https://n.neurology.org/content/92/15_Supplement/S25.007 9 June 2021閲覧。.
↑  “2021年03月19日｜ロシュ社、2～25歳のII型またはIII型脊髄性筋萎縮症（SMA）において運動機能の改善・維持を示すEvrysdiTM（リスジプラム）の2年データを発表｜ニュースリリース｜中外製薬”. 中外製薬企業情報サイト. 2021年10月18日閲覧。
↑  “2021年04月21日｜Evrysdi（リスジプラム）、乳児のI型脊髄性筋萎縮症（SMA）において継続して運動機能および生存の改善・維持を示す｜ニュースリリース｜中外製薬”. 中外製薬企業情報サイト. 2021年10月18日閲覧。
↑  Inacio, Patricia (21 December 2018). “Risdiplam Granted EMA's PRIME Designation for Potential in Spinal Muscular Atrophy” (English). SMA News Today (Pensacola, Florida, United States: BioNews Services (BioNews Services, LLC.)).  オリジナルの26 January 2021時点におけるアーカイブ。. https://web.archive.org/web/20210126185434/https://smanewstoday.com/news-posts/2018/12/21/risdiplam-granted-ema-prime-designation 9 June 2021閲覧。
↑  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). pp. 1–4. 2020年5月6日時点のオリジナルよりアーカイブ (PDF)。
↑  “2019年03月27日｜リスジプラムが脊髄性筋萎縮症に対する希少疾病用医薬品に指定｜ニュースリリース｜中外製薬”. 中外製薬企業情報サイト. 2021年10月18日閲覧。
↑  PTC Therapeutics (17 August 2020). “PTC Announces the Acceptance of the European Marketing Authorization Application for Evrysdi™ (risdiplam) for the Treatment of Spinal Muscular Atrophy”. PTC Therapeutics. Albuquerque, New Mexico, United States of America: PR Newswire.  オリジナルの18 August 2020時点におけるアーカイブ。. https://web.archive.org/web/20200818182239/https://www.prnewswire.com/news-releases/ptc-announces-the-acceptance-of-the-european-marketing-authorization-application-for-evrysdi-risdiplam-for-the-treatment-of-spinal-muscular-atrophy-301113095.html 9 June 2021閲覧。