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Article History

Received: 21 April 2025

Accepted: 24 June 2025

First Online: 9 August 2025

Declarations

:

: 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.

: 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—immediate family member, Inst—my 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’ 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’ 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’ 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’ 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’ bureaus, manuscript writing or educational events (Chugai Pharmaceutical Co., Ltd.). Yuka Ishikawa: Consulting fees (Kaneka Corporation); payment or honoraria for lectures, presentations, speakers’ 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’ bureaus, manuscript writing or educational events (Biogen Japan; Chugai Pharmaceutical Co., Ltd.). Takahiko Tsumuraya, Hiromitsu Kawata and Yuki Miyano are employed by Chugai Pharmaceutical Co., Ltd. Hirofumi Komaki: Consulting fees (Roche); payment or honoraria for lectures, presentations, speakers’ bureaus, manuscript writing or educational events (Chugai Pharmaceutical Co., Ltd.; Novartis Pharmaceutical Co., Ltd.); support for attending meetings and/or travel (Roche).

: 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.