Goldberg, Yigal Paul
Navon-Perry, Leehee
Cruz-Herranz, Andrés
Chen, Kelly
Hecker-Barth, Gabriele
Spiegel, Katrin
Cohen, Yael
Niethammer, Martin
Tan, Andrew M. https://orcid.org/0000-0002-0099-8814
Schuring, Henk
Geva, Michal
Hayden, Michael R.
Article History
Accepted: 11 February 2025
First Online: 7 March 2025
Declarations
:
: The authors have no funding to report. Open access funding was provided by Prilenia Therapeutics B.V.
: L.N.P., A.C.H., K.C., A.M.T., Y.C., H.S., and M.G. are employees of Prilenia Therapeutics B.V. and may have stock options in the company. M.R.H. is the CEO and scientific co-founder of Prilenia Neurotherapeutics B.V. He is also a Physician Scientist and University Killam Professor at the University of British Columbia. M.R.H. serves on the Board of Directors for Ionis Pharmaceuticals (San Diego), 89Bio (San Francisco), AbCellera (Vancouver), and Oxford Biomedica (Oxford) (2022–2024). Y.P.G. has stock options in Prilenia. The remaining authors declare no conflicts of interest related to this work.
: Not applicable.
: Not applicable.
: Not applicable.
: The data supporting the findings of this study are based on an integrated safety analysis of pooled data from 1067 patients across four double-blind, placebo-controlled clinical trials: PROOF-HD (NCT04556656, EudraCT no. 2020-002822-10), PRIDE-HD (NCT02006472, EudraCT no. 2013-001888-23), MermaiHD (NCT00665223, EudraCT no. 2007-004988-22), and HART (NCT00724048). Data requests for these trials can be requested from the corresponding author, subject to ethical and regulatory considerations.
: Not applicable.
: The study was conceived by Y.P.G., M.G., M.N., M.R.H., and Y.C., who also contributed to writing and developing the manuscript. L.N.P., A.C.H., K.S., A.M.T., and H.S. contributed to writing and editing, with K.S. also providing intellectual and editorial support. K.C. handled data analysis and management support, while M.N. and M.R.H. provided further guidance.