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Authors
Numajiri, Naoki https://orcid.org/0009-0007-9873-721X
Yamamoto, Takanori
Waratani, Miina https://orcid.org/0000-0002-4648-7454
Takahashi, Masanori P. https://orcid.org/0000-0002-5513-5012
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Funding
Funding for this research was provided by:
Alexion Pharma GK, AstraZeneca Rare Disease
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Article History
Received: 23 July 2025
Accepted: 21 October 2025
First Online: 12 November 2025
Declarations
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: Naoki Numajiri is an employee of and holds stock or stock options in Alexion Pharma GK, AstraZeneca Rare Disease. Takanori Yamamoto is an employee of Alexion Pharma GK, AstraZeneca Rare Disease. Miina Waratani is a former employee of and holds stock or stock options in AstraZeneca. Masanori P. Takahashi reports unrestricted research grants from Astellas Pharma, Japan Blood Products Organization, and Mitsubishi Tanabe Pharma, outside of the submitted work, and honoraria from argenx, Alexion Pharmaceuticals, Immunovant, and UCB Pharma.
: This study was conducted in accordance with the Declaration of Helsinki: Ethical Principles for Medical Research Involving Human Participants and all applicable laws and regulations in Japan. The study protocol was approved by Alexion’s Health Economics and Outcomes Research Review Committee. It was not necessary or practical to obtain informed consent for the use of secondary data in this study; however, all data in the JMDC was de-identified per the Act on the Protection of Personal Information, and no personal information could be restored in the database and no individual could be re-identified.
