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Article History

Received: 29 July 2025

Accepted: 6 October 2025

First Online: 4 November 2025

Declarations

:

: The clinical trials CONVERT and INS-312 are registered at ClinicalTrials.gov (registration numbers NCT02344004 and NCT02628600, respectively). The studies were approved at all institutions, and the IRBs for the sites that enrolled patients included in this case series were Keio University Hospital IRB (chair person, Daisuke Aoki, MD, PhD), National Hospital Organization Higashi Nagoya National Hospital IRB (chair person, Kenji Ogawa, MD, PhD), and Review Board of Human Rights and Ethics for Clinical Studies IRB (chairperson, Norihiko Basugi, MD, PhD). Collection of information from patient charts and the clinical trial reports for the patients included in this case series were conducted in accordance with the Declaration of Helsinki. All patients provided informed consent prior to participation in the CONVERT and INS-312 trials. Written informed consent was obtained from the two patients for whom additional investigator collected data were included and for the publication of this data. Additional data for patients 3 and 4 were not presented because informed consent was not received.

: Yoshitaka Yamazaki has nothing to disclose. Taku Nakagawa received lecture fees from Insmed Incorporated and was a member of an advisory board of Insmed Incorporated. Zhanna Jumadilova is a former employee of Insmed Incorporated and received grants or contracts from Insmed Incorporated. Their current affiliation is Pfizer, 66 Hudson Blvd E, New York, NY 10001, USA. Dayton Yuen and Raul Villa are employees and shareholders of Insmed Incorporated and report stock or stock options in Insmed Incorporated. Naoki Hasegawa serves as a consultant to Insmed Incorporated. His current affiliation is Yokohama Municipal Citizen’s Hospital, 1–1 Mitsuzawa Nishimachi, Kanagawa-ku, Yokohama-shi, Kanagawa 221–0855, Japan.