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Authors
Cappelli, Francesco https://orcid.org/0000-0002-1622-0732
Ponti, Lucia
Smorti, Martina
Hsu, Kristen
Damy, Thibaud
Verheyen, Nicolas
Wang, Ronnie
Kumar, Nisith
Munteanu, Carmen
,
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Funding
Funding for this research was provided by:
Pfizer
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Article History
Received: 20 August 2025
Accepted: 28 October 2025
First Online: 18 December 2025
Declarations
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: Francesco Cappelli reports honoraria for advisory board participation from Alnylam, Astra Zeneca, Bayer, BridgeBio, Novo Nordisk, and Pfizer; his institution has received an unconditional research grant from Pfizer. Martina Smorti reports speaker fees from Pfizer. Lucia Ponti and Kristen Hsu have no disclosures. Thibaud Damy has received consulting fees from Alnylam, GlaxoSmithKline, Pfizer, and Prothena; honoraria from Alnylam, Pfizer, and Prothena; research grants from GlaxoSmithKline and Pfizer; and clinical trial support from Alnylam, Ionis, and Pfizer. Nicolas Verheyen has received research grant, honoraria for advisory board participation, and speaker’s fees from Pfizer. Ronnie Wang, Nisith Kumar, and Carmen Munteanu are employees of Pfizer and hold stock/stock options.
: Patients and caregivers provided informed consent to participate in the study. The study was approved by the institutional review board or independent ethics committee at each site. The study was conducted in accordance with the Declaration of Helsinki.
