aus dem Siepen, Fabian https://orcid.org/0000-0003-4264-1722
Dispenzieri, Angela https://orcid.org/0000-0001-8780-9512
Hanna, Mazen https://orcid.org/0000-0002-7251-3003
Carlsson, Martin
Garcia-Pavia, Pablo https://orcid.org/0000-0002-5470-2257
Funding for this research was provided by:
Envision Pharma Group
Article History
Received: 27 November 2025
Accepted: 9 July 2026
First Online: 27 August 2026
Declarations
Conflicts of Interest: Fabian aus dem Siepen reports speaker fees from Pfizer, Ionis Pharmaceuticals, Alnylam Pharmaceuticals, and Janssen, and consulting fees from Pfizer and Akcea Therapeutics. Angela Dispenzieri reports serving on advisory boards and independent review committees for Janssen; research funding from Alynlam, Pfizer, Takeda, BMS, and AbbVie; and serving on a scientific advisory board for HemalogiX. Mazen Hanna reports serving on advisory boards for Alnylam, Eidos, Ionis, Alexion Pharmaceuticals, and Pfizer. Martin Carlsson is an employee of Pfizer and holds stock/stock options in Pfizer. Pablo Garcia-Pavia reports speaker fees from Alnylam Pharmaceuticals, AstraZeneca, Bayer, BridgeBio, Intellia, Ionis Pharmaceuticals, Novo Nordisk, and Pfizer; consulting fees from Alexion, Alnylam Pharmaceuticals, AstraZeneca, Attralus, Bayer, BridgeBio, Intellia, Ionis Pharmaceuticals, Neurimmune, Novo Nordisk, and Pfizer; and research/educational support to his institution from Alnylam Pharmaceuticals, AstraZeneca, BridgeBio, Intellia, Novo Nordisk, and Pfizer.
Ethical Approval: Both studies were approved by the independent review board or ethics committee at each participating center. These studies were conducted in accordance with the concepts laid out in the Declaration of Helsinki and the International Conference on Harmonisation Good Clinical Practice guidelines. All patients provided written informed consent.