{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,25]],"date-time":"2026-09-25T15:48:31Z","timestamp":1790351311334,"version":"4.1.0"},"reference-count":35,"publisher":"Springer Science and Business Media LLC","issue":"5","license":[{"start":{"date-parts":[[2025,8,9]],"date-time":"2025-08-09T00:00:00Z","timestamp":1754697600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"},{"start":{"date-parts":[[2025,8,9]],"date-time":"2025-08-09T00:00:00Z","timestamp":1754697600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"}],"funder":[{"DOI":"10.13039\/100010795","name":"Chugai Pharmaceutical Co., Ltd.","doi-asserted-by":"crossref","id":[{"id":"10.13039\/100010795","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Neurol Ther"],"published-print":{"date-parts":[[2025,10]]},"DOI":"10.1007\/s40120-025-00795-x","type":"journal-article","created":{"date-parts":[[2025,8,9]],"date-time":"2025-08-09T14:44:34Z","timestamp":1754750674000},"page":"2083-2094","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Safety of Risdiplam in Japanese Patients with Spinal Muscular Atrophy: A 12\u2011Month Interim Analysis of a Postmarketing Surveillance Study"],"prefix":"10.1007","volume":"14","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2632-0873","authenticated-orcid":false,"given":"Kayoko","family":"Saito","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0432-0402","authenticated-orcid":false,"given":"Toshio","family":"Saito","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0910-8910","authenticated-orcid":false,"given":"Reiko","family":"Arakawa","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6468-7185","authenticated-orcid":false,"given":"Yasuhiro","family":"Takeshima","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6778-4511","authenticated-orcid":false,"given":"Hisahide","family":"Nishio","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1406-9768","authenticated-orcid":false,"given":"Yuka","family":"Ishikawa","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9453-9311","authenticated-orcid":false,"given":"Masahisa","family":"Katsuno","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0009-7261-0346","authenticated-orcid":false,"given":"Takahiko","family":"Tsumuraya","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0007-4018-6945","authenticated-orcid":false,"given":"Hiromitsu","family":"Kawata","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9964-6690","authenticated-orcid":false,"given":"Yuki","family":"Miyano","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0659-1417","authenticated-orcid":false,"given":"Hirofumi","family":"Komaki","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,8,9]]},"reference":[{"key":"795_CR1","unstructured":"Prior TW, Leach ME, Finanger E. Spinal muscular atrophy. In: Adam MP, Feldman J, Mirzaa GM, et al., eds. GeneReviews\u00ae. Seattle: University of Washington; 1993\u20132024. https:\/\/www.ncbi.nlm.nih.gov\/books\/NBK1352\/."},{"key":"795_CR2","doi-asserted-by":"crossref","unstructured":"Wilton-Clark H, Al-Aghbari A, Yang J, Yokota T. Advancing epidemiology and genetic approaches for the treatment of spinal and bulbar muscular atrophy: focus on prevalence in the indigenous population of western Canada. Genes (Basel). 2023;14(8):1634.","DOI":"10.3390\/genes14081634"},{"key":"795_CR3","doi-asserted-by":"crossref","unstructured":"Nishio H, Niba ETE, Saito T, Okamoto K, Takeshima Y, Awano H. Spinal muscular atrophy: the past, present, and future of diagnosis and treatment. Int J Mol Sci. 2023;24(15):11939.","DOI":"10.3390\/ijms241511939"},{"issue":"1","key":"795_CR4","doi-asserted-by":"publisher","first-page":"2","DOI":"10.1016\/j.braindev.2021.08.002","volume":"44","author":"M Ito","year":"2022","unstructured":"Ito M, Yamauchi A, Urano M, et al. Epidemiological investigation of spinal muscular atrophy in Japan. Brain Dev. 2022;44(1):2\u201316.","journal-title":"Brain Dev"},{"issue":"1","key":"795_CR5","doi-asserted-by":"publisher","first-page":"300","DOI":"10.1186\/s13023-022-02455-x","volume":"17","author":"C Cances","year":"2022","unstructured":"Cances C, Vlodavets D, Comi GP, et al. Natural history of Type 1 spinal muscular atrophy: a retrospective, global, multicenter study. Orphanet J Rare Dis. 2022;17(1):300.","journal-title":"Orphanet J Rare Dis"},{"issue":"1","key":"795_CR6","doi-asserted-by":"publisher","first-page":"449","DOI":"10.1007\/s40120-021-00307-7","volume":"11","author":"E Viscidi","year":"2022","unstructured":"Viscidi E, Juneja M, Wang J, et al. Comparative all-cause mortality among a large population of patients with spinal muscular atrophy versus matched controls. Neurol Ther. 2022;11(1):449\u201357.","journal-title":"Neurol Ther"},{"issue":"1","key":"795_CR7","doi-asserted-by":"publisher","first-page":"52","DOI":"10.1038\/s41572-022-00380-8","volume":"8","author":"E Mercuri","year":"2022","unstructured":"Mercuri E, Sumner CJ, Muntoni F, Darras BT, Finkel RS. Spinal muscular atrophy. Nat Rev Dis Primers. 2022;8(1):52.","journal-title":"Nat Rev Dis Primers"},{"issue":"18","key":"795_CR8","doi-asserted-by":"publisher","first-page":"1723","DOI":"10.1056\/NEJMoa1702752","volume":"377","author":"RS Finkel","year":"2017","unstructured":"Finkel RS, Mercuri E, Darras BT, et al. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med. 2017;377(18):1723\u201332.","journal-title":"N Engl J Med"},{"issue":"7","key":"795_CR9","doi-asserted-by":"publisher","first-page":"625","DOI":"10.1056\/NEJMoa1710504","volume":"378","author":"E Mercuri","year":"2018","unstructured":"Mercuri E, Darras BT, Chiriboga CA, et al. Nusinersen versus sham control in later-onset spinal muscular atrophy. N Engl J Med. 2018;378(7):625\u201335.","journal-title":"N Engl J Med"},{"key":"795_CR10","unstructured":"Pharmaceuticals and Medical Devices Agency. List of approved products\u2014new drugs. https:\/\/www.pmda.go.jp\/english\/review-services\/reviews\/approved-information\/drugs\/0002.html. Accessed Jan 2025."},{"issue":"4","key":"795_CR11","doi-asserted-by":"publisher","first-page":"284","DOI":"10.1016\/S1474-4422(21)00001-6","volume":"20","author":"JW Day","year":"2021","unstructured":"Day JW, Finkel RS, Chiriboga CA, et al. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial. Lancet Neurol. 2021;20(4):284\u201393.","journal-title":"Lancet Neurol"},{"issue":"10","key":"795_CR12","doi-asserted-by":"publisher","first-page":"832","DOI":"10.1016\/S1474-4422(21)00251-9","volume":"20","author":"E Mercuri","year":"2021","unstructured":"Mercuri E, Muntoni F, Baranello G, et al. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial. Lancet Neurol. 2021;20(10):832\u201341.","journal-title":"Lancet Neurol"},{"key":"795_CR13","unstructured":"Pharmaceuticals and Medical Devices Agency. List of approved products\u2014new regenerative medical products. https:\/\/www.pmda.go.jp\/english\/review-services\/reviews\/approved-information\/0002.html. Accessed Jan 2025."},{"issue":"6","key":"795_CR14","doi-asserted-by":"publisher","first-page":"753","DOI":"10.1111\/dmcn.15135","volume":"64","author":"KA Carey","year":"2022","unstructured":"Carey KA, Farrar MA, Kasparian NA, Street DJ, De Abreu LR. Family, healthcare professional, and societal preferences for the treatment of infantile spinal muscular atrophy: a discrete choice experiment. Dev Med Child Neurol. 2022;64(6):753\u201361.","journal-title":"Dev Med Child Neurol"},{"issue":"3","key":"795_CR15","doi-asserted-by":"publisher","first-page":"305","DOI":"10.1002\/ana.26299","volume":"91","author":"CJJ Yeo","year":"2022","unstructured":"Yeo CJJ, Simmons Z, De Vivo DC, Darras BT. Ethical perspectives on treatment options with spinal muscular atrophy patients. Ann Neurol. 2022;91(3):305\u201316.","journal-title":"Ann Neurol"},{"issue":"15","key":"795_CR16","doi-asserted-by":"publisher","first-page":"6501","DOI":"10.1021\/acs.jmedchem.8b00741","volume":"61","author":"H Ratni","year":"2018","unstructured":"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\u201317.","journal-title":"J Med Chem"},{"issue":"4","key":"795_CR17","doi-asserted-by":"publisher","first-page":"401","DOI":"10.1007\/s40263-022-00910-8","volume":"36","author":"J Paik","year":"2022","unstructured":"Paik J. Risdiplam: a review in spinal muscular atrophy. CNS Drugs. 2022;36(4):401\u201310.","journal-title":"CNS Drugs"},{"issue":"5","key":"795_CR18","doi-asserted-by":"publisher","first-page":"427","DOI":"10.1056\/NEJMoa2102047","volume":"385","author":"BT Darras","year":"2021","unstructured":"Darras BT, Masson R, Mazurkiewicz-Be\u0142dzi\u0144ska M, et al. Risdiplam-treated infants with type 1 spinal muscular atrophy versus historical controls. N Engl J Med. 2021;385(5):427\u201335.","journal-title":"N Engl J Med"},{"issue":"7","key":"795_CR19","doi-asserted-by":"publisher","first-page":"1945","DOI":"10.1111\/ene.15499","volume":"30","author":"E Mercuri","year":"2023","unstructured":"Mercuri E, Baranello G, Boespflug-Tanguy O, et al. Risdiplam in types 2 and 3 spinal muscular atrophy: a randomised, placebo-controlled, dose-finding trial followed by 24\u00a0months of treatment. Eur J Neurol. 2023;30(7):1945\u201356.","journal-title":"Eur J Neurol"},{"issue":"5","key":"795_CR20","doi-asserted-by":"publisher","first-page":"2531","DOI":"10.1007\/s00415-023-11560-1","volume":"270","author":"M Oskoui","year":"2023","unstructured":"Oskoui M, Day JW, Deconinck N, et al. Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA). J Neurol. 2023;270(5):2531\u201346.","journal-title":"J Neurol"},{"key":"795_CR21","unstructured":"U.S. Food and Drug Administration. EVRYSDI\u00ae (risdiplam) for oral solution. https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2022\/213535s003s005lbl.pdf. Accessed Jan 2025."},{"issue":"12","key":"795_CR22","doi-asserted-by":"publisher","first-page":"1110","DOI":"10.1016\/S1474-4422(22)00339-8","volume":"21","author":"R Masson","year":"2022","unstructured":"Masson R, Mazurkiewicz-Be\u0142dzi\u0144ska M, Rose K, et al. Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial. Lancet Neurol. 2022;21(12):1110\u20139.","journal-title":"Lancet Neurol"},{"issue":"1","key":"795_CR23","doi-asserted-by":"publisher","first-page":"42","DOI":"10.1016\/S1474-4422(21)00367-7","volume":"21","author":"E Mercuri","year":"2022","unstructured":"Mercuri E, Deconinck N, Mazzone ES, et al. Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial. Lancet Neurol. 2022;21(1):42\u201352.","journal-title":"Lancet Neurol"},{"key":"795_CR24","unstructured":"International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH). ICH harmonised tripartite guideline. Post-approval safety data management: definitions and standards for expedited reporting E2D. https:\/\/www.pmda.go.jp\/files\/000156499.pdf. Accessed May 2025."},{"key":"795_CR25","unstructured":"Japan Pharmaceutical Manufacturers Association. Post-marketing surveillance of drugs. https:\/\/www.jpma.or.jp\/english\/about\/parj\/eki4g6000000784o-att\/2020e_ch04.pdf. Accessed Jan 2025."},{"issue":"1","key":"795_CR26","doi-asserted-by":"publisher","first-page":"258","DOI":"10.1186\/s13023-023-02872-6","volume":"18","author":"DG Whitney","year":"2023","unstructured":"Whitney DG, Knierbein EEN, Daunter AK. Prevalence of morbidities across the lifespan for adults with spinal muscular atrophy: a retrospective cohort study. Orphanet J Rare Dis. 2023;18(1):258.","journal-title":"Orphanet J Rare Dis"},{"issue":"8","key":"795_CR27","doi-asserted-by":"publisher","first-page":"4871","DOI":"10.1007\/s00415-024-12318-z","volume":"271","author":"CA Chiriboga","year":"2024","unstructured":"Chiriboga CA, Bruno C, Duong T, et al. JEWELFISH: 24-month results from an open-label study in non-treatment-na\u00efve patients with SMA receiving treatment with risdiplam. J Neurol. 2024;271(8):4871\u201384.","journal-title":"J Neurol"},{"issue":"2","key":"795_CR28","doi-asserted-by":"publisher","first-page":"543","DOI":"10.1007\/s40120-023-00444-1","volume":"12","author":"CA Chiriboga","year":"2023","unstructured":"Chiriboga CA, Bruno C, Duong T, et al. Risdiplam in patients previously treated with other therapies for spinal muscular atrophy: an interim analysis from the JEWELFISH Study. Neurol Ther. 2023;12(2):543\u201357.","journal-title":"Neurol Ther"},{"issue":"10","key":"795_CR29","doi-asserted-by":"publisher","first-page":"915","DOI":"10.1056\/NEJMoa2009965","volume":"384","author":"G Baranello","year":"2021","unstructured":"Baranello G, Darras BT, Day JW, et al. Risdiplam in type 1 spinal muscular atrophy. N Engl J Med. 2021;384(10):915\u201323.","journal-title":"N Engl J Med"},{"issue":"3","key":"795_CR30","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0298609","volume":"19","author":"L Yu","year":"2024","unstructured":"Yu L, Liu L. Exploration of adverse events associated with risdiplam use: retrospective cases from the US Food and Drug Administration Adverse Event Reporting System (FAERS) database. PLoS ONE. 2024;19(3):e0298609.","journal-title":"PLoS ONE"},{"issue":"12","key":"795_CR31","doi-asserted-by":"publisher","first-page":"3501","DOI":"10.1007\/s11999-011-2080-y","volume":"469","author":"JH Yang","year":"2011","unstructured":"Yang JH, Kasat NS, Suh SW, Kim SY. Improvement in reflux gastroesophagitis in a patient with spinal muscular atrophy after surgical correction of kyphoscoliosis: a case report. Clin Orthop Relat Res. 2011;469(12):3501\u20135.","journal-title":"Clin Orthop Relat Res"},{"key":"795_CR32","doi-asserted-by":"crossref","unstructured":"Kimizu T, Ida S, Okamoto K, et al. Spinal muscular atrophy: diagnosis, incidence, and newborn screening in Japan. Int J Neonatal Screen. 2021;7(3):45.","DOI":"10.3390\/ijns7030045"},{"issue":"1","key":"795_CR33","doi-asserted-by":"publisher","first-page":"36","DOI":"10.1016\/j.braindev.2018.07.016","volume":"41","author":"K Okamoto","year":"2019","unstructured":"Okamoto K, Fukuda M, Saito I, et al. Incidence of infantile spinal muscular atrophy on Shikoku Island of Japan. Brain Dev. 2019;41(1):36\u201342.","journal-title":"Brain Dev"},{"issue":"12","key":"795_CR34","doi-asserted-by":"publisher","first-page":"2211","DOI":"10.3390\/genes14122211","volume":"14","author":"S Sonehara","year":"2023","unstructured":"Sonehara S, Bo R, Nambu Y, et al. Newborn screening for spinal muscular atrophy: a 2.5-year experience in Hyogo Prefecture, Japan. Genes (Basel). 2023;14(12):2211.","journal-title":"Genes (Basel)"},{"issue":"1","key":"795_CR35","doi-asserted-by":"publisher","DOI":"10.1212\/CPJ.0000000000200224","volume":"14","author":"VD Armengol","year":"2024","unstructured":"Armengol VD, Darras BT, Abulaban AA, et al. Life-saving treatments for spinal muscular atrophy: global access and availability. Neurol Clin Pract. 2024;14(1):e200224.","journal-title":"Neurol Clin Pract"}],"container-title":["Neurology and Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40120-025-00795-x.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s40120-025-00795-x\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40120-025-00795-x.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,9,20]],"date-time":"2025-09-20T11:22:18Z","timestamp":1758367338000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s40120-025-00795-x"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,8,9]]},"references-count":35,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2025,10]]}},"alternative-id":["795"],"URL":"https:\/\/doi.org\/10.1007\/s40120-025-00795-x","relation":{},"ISSN":["2193-8253","2193-6536"],"issn-type":[{"value":"2193-8253","type":"print"},{"value":"2193-6536","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,8,9]]},"assertion":[{"value":"21 April 2025","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"24 June 2025","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"9 August 2025","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"Kayoko Saito was also the Principal Investigator for clinical trials of Chugai (Roche) (SUNFISH), Biogen (CHERISH, ENDEAR, DEVOTE) and Novartis Gene Therapies, Inc.\/Novartis (SPR1NT, LT-002, STRENGTH), during the time this study was conducted. All these trials also involved patients with SMA. Payments were made to the university hospital for these trials. Reiko Arakawa was also the Principal Investigator for clinical trials of Chugai (Roche) (MANATEE, SUNFISH), and the Sub Investigator for clinical trials of Biogen (CHERISH, ENDEAR) during the time this study was conducted. All these trials also involved patients with SMA. Payments were made to the hospital for these trials.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of Interest"}},{"value":"The following represents disclosure information provided by authors of this manuscript: All relationships are considered compensated unless otherwise noted. Relationships are self-held unless noted. I\u2014immediate family member, Inst\u2014my institution. Relationships may not relate to the subject matter of this manuscript. Kayoko Saito: Grants or contracts from any entity (Biogen Japan [Inst]; National grant of Japan for AMED [Grant number: 20ek0109472h0001, 21ek0109472h0002, 22ek0109472h0003] and the MHLW Research in Rare and Intractable Diseases Program [Grant Number: JPMH23FC1008]); payment or honoraria for lectures, presentations, speakers\u2019 bureaus, manuscript writing or educational events (Biogen Japan; Chugai Pharmaceutical Co., Ltd.; Novartis Pharmaceutical Co., Ltd); participation on a Data Safety Monitoring Board or Advisory Board (Biogen Japan; Chugai Pharmaceutical Co., Ltd.; Novartis Pharmaceutical Co., Ltd); leadership or fiduciary role in other boards, societies, committees or advocacy groups, paid or unpaid (medical adviser of the Japan SMA Families Network). Toshio Saito: Payment or honoraria for lectures, presentations, speakers\u2019 bureaus, manuscript writing or educational events (Biogen Japan; Chugai Pharmaceutical Co., Ltd.; payment for expert testimony (Biogen Japan; Chugai Pharmaceutical Co., Ltd.); participation on a Data Safety Monitoring Board or Advisory Board (Biogen Japan; Chugai Pharmaceutical Co., Ltd.). Reiko Arakawa: Consulting fees (Chugai Pharmaceutical Co., Ltd.); payment or honoraria for lectures, presentations, speakers\u2019 bureaus, manuscript writing or educational events (Biogen Japan; Chugai Pharmaceutical Co., Ltd.; Novartis Pharmaceutical Co., Ltd.; Roche Korea Co., Ltd.); participation on a Data Safety Monitoring Board or Advisory Board (Biogen Japan; Chugai Pharmaceutical Co., Ltd.; Novartis Pharmaceutical Co., Ltd.). Yasuhiro Takeshima: Payment or honoraria for lectures, presentations, speakers\u2019 bureaus, manuscript writing or educational events (Biogen Japan; Chugai Pharmaceutical Co., Ltd.; Novartis Pharmaceutical Co., Ltd.). Hisahide Nishio: Payment or honoraria for lectures, presentations, speakers\u2019 bureaus, manuscript writing or educational events (Chugai Pharmaceutical Co., Ltd.). Yuka Ishikawa: Consulting fees (Kaneka Corporation); payment or honoraria for lectures, presentations, speakers\u2019 bureaus, manuscript writing or educational events (Biogen Co.; Breas Co.; Cough Ventec Co.; Chugai Co.; Chest Co.; Nihon Shinyaku Co; Philips Japan); support for attending meetings and\/or travel (Chugai Co.; Cough Ventec Co.). Masahisa Katsuno: Consulting fees (Chugai Pharmaceutical Co., Ltd.); research grants (Biogen Japan; Chugai Pharmaceutical Co., Ltd.); payment or honoraria for lectures, presentations, speakers\u2019 bureaus, manuscript writing or educational events (Biogen Japan; Chugai Pharmaceutical Co., Ltd.). Takahiko Tsumuraya, Hiromitsu Kawata\u00a0and Yuki Miyano are employed by Chugai Pharmaceutical Co., Ltd. Hirofumi Komaki: Consulting fees (Roche); payment or honoraria for lectures, presentations, speakers\u2019 bureaus, manuscript writing or educational events (Chugai Pharmaceutical Co., Ltd.; Novartis Pharmaceutical Co., Ltd.); support for attending meetings and\/or travel (Roche).","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Financial Disclosures for the Previous 36 Months"}},{"value":"The study did not undergo review by the ethics committee of the participating medical institutions nor follow procedures for informed consent, as these were not required for PMS studies according to Japanese regulations under the Act on Securing Quality, Efficacy, and Safety of Products Including Pharmaceuticals and Medical Devices []. This was necessary to ensure the registration of all patients with SMA and all patients were included as part of the approval conditions in Japan. Patient data were collected after identification. The study was conducted in accordance with relevant regulations in Japan (Ministerial Ordinance on Good Post-Marketing Study Practice [GPSP]; Ministry of Health, Labour and Welfare Ordinance Number 38; 23 March 2005). The study protocol was reviewed and approved by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) prior to study initiation.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical Approval"}}]}}