Zhang, Steven https://orcid.org/0009-0007-3555-8011
Gamallo, Pablo https://orcid.org/0000-0002-2429-0984
Rawson, Verity
Funding for this research was provided by:
Novo Nordisk
Article History
Accepted: 5 June 2025
First Online: 2 July 2025
Declarations
:
: This study was supported by Dicerna Pharmaceuticals, Inc., a Novo Nordisk Company (Lexington, MA, USA).
: All authors are employees, and SZ and VR are shareholders of Novo Nordisk.
: The institutional review board or ethics committee at each participating center approved the final study protocols.
: Written informed consent was obtained from all adult participants and participating children’s parents or legal guardians. The safety review committee convened at predefined decision points and the occurrence of any potential dose-limiting toxicities to ensure the acceptability of continued nedosiran administration within each study (and cohort) and of dose escalation to subsequent cohorts (where applicable).
: Not applicable.
: Data will be made available, on reasonable request.
: Not applicable.
: Steven Zhang was responsible for the study conception and design. Model development and simulations were performed by Pablo Gamallo. Verity Rawson ensured medical accuracy. All authors participated in the interpretation of the study data, drafting of the manuscript, critical revision, and approval of the final version of the manuscript.