Document is current

Article History

Received: 24 April 2026

Accepted: 13 May 2026

First Online: 8 June 2026

Declarations

:

: a) Funding: This work received no external or internal funding. b) Disclosures of interest IK has participated in a research grant to Imperial College from Ultragenyx. KBH reports lecture fees/honoraria from Ultragenyx, Menarini, Amgen, and Sanofi. KKR reports unrestricted research grants (last 3 years) to Imperial College London from Amgen, Sanofi, Regeneron, Daiichi Sankyo, and Ultragenyx; Consulting fees from Novartis, Daiichi Sankyo, Kowa, Esperion, Novo Nordisk, MSD, Lilly, Silence Therapeutics, AZ, New Amsterdam Pharma, Bayer, Beren Therapeutics, CLEERLY, EMENDOBIO, SCRIBE, CRISPR, VAXXINITY, Amarin, Regeneron, Ultragenix, Cargene, Resverlogix for serving as a member of the SC or EC of clinical trials and roles as PI, NLI. Attending advisory boards, providing advice on data, its interpretation and future lines of research; lecture fees from Novartis, BI, AZ, Novo Nordisk, Viatris, Amarin, Biologix Pharma, Sanofi, Amgen, Esperion, Daiichi Sankyo, Macleod Pharma for CME and non-CME symposia at international meetings and stock options from New Amsterdam Pharma and PEMI31. AE has participated in research grants to Imperial College from Amgen, Regeneron, Daiichi Sankyo, and Ultragenyx. c) Human and animal rights and informed consent This review does not contain any studies with human or animal subjects performed by any of the authors.

: This review does not contain any studies with human or animal subjects performed by any of the authors.