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Authors
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Lee, Jong-Min https://orcid.org/0000-0001-5799-0787
McLean, Zachariah L.
Correia, Kevin
Shin, Jun Wan
Lee, Sujin
Jang, Jae-Hyun https://orcid.org/0009-0005-9330-3346
Lee, Yukyeong https://orcid.org/0000-0003-4569-7377
Kim, Kyung-Hee
Choi, Doo Eun
Long, Jeffrey D.
Lucente, Diane
Seong, Ihn Sik
Mouro Pinto, Ricardo https://orcid.org/0000-0001-6744-2805
Giordano, James V.
Mysore, Jayalakshmi S.
Siciliano, Jacqueline
Elezi, Emanuela
Ruliera, Jayla
Gillis, Tammy
Wheeler, Vanessa C. https://orcid.org/0009-0004-8259-5796
MacDonald, Marcy E.
Gusella, James F. https://orcid.org/0000-0003-0681-9263
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Gatseva, Anna https://orcid.org/0000-0003-1601-5277
Ciosi, Marc https://orcid.org/0000-0002-7663-4080
Lomeikaite, Vilija
Loay, Hossameldin https://orcid.org/0000-0001-9145-1736
Monckton, Darren G. https://orcid.org/0000-0002-8298-8264
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Wills, Christopher https://orcid.org/0000-0002-3178-8924
Massey, Thomas H. https://orcid.org/0000-0002-9804-2131
Jones, Lesley
Holmans, Peter https://orcid.org/0000-0003-0870-9412
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Kwak, Seung https://orcid.org/0000-0003-4048-1278
Sampaio, Cristina
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Orth, Michael
Landwehrmeyer, G. Bernhard
Paulsen, Jane S.
Dorsey, E. Ray
Myers, Richard H. https://orcid.org/0000-0002-8365-2674
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Funding
Funding for this research was provided by:
CHDI Foundation (A-2690)
U.S. Department of Health & Human Services | NIH | National Institute of Neurological Disorders and Stroke (NS082079, NS016367, NS114065, NS126420, NS049206, NS127866, NS105709, NS119471)
Hereditary Disease Foundation
RCUK | Medical Research Council (MR/X018253/1, MR/L010305/1)
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Article History
Received: 23 July 2024
Accepted: 11 April 2025
First Online: 9 June 2025
Competing interests
: J.F.G. and V.C.W. were founding scientific advisory board members with a financial interest in Triplet Therapeutics. Their financial interests were reviewed and are managed by Massachusetts General Hospital (MGH) and MGB in accordance with their conflict of interest policies. J.F.G. consults for Transine Therapeutics (dba Harness Therapeutics), has previously provided paid consulting services to Wave Therapeutics USA, Biogen and Pfizer, and receives research funding from Pfizer. V.C.W. is a scientific advisory board member of LoQus23 Therapeutics and has provided paid consulting services to Acadia Pharmaceuticals, Alnylam, Biogen, Passage Bio and Rgenta Therapeutics. V.C.W. and R.M.P. have received research support from Pfizer. J.-M.L. consults for GenKOre and serves on the scientific advisory board of GenEdit. Within the last 36 months, D.G.M. has been a scientific consultant and/or received an honoraria or grants from AMO Pharma, Dyne, F. Hoffman–La Roche, LoQus23, MOMA Therapeutics, Novartis, Ono Pharmaceuticals, Pfizer Pharmaceuticals, Rgenta Therapeutics, Sanofi, Sarepta Therapeutics, Script Biosciences, Triplet Therapeutics and Vertex Pharmaceuticals. D.G.M. also had research contracts with AMO Pharma and Vertex Pharmaceuticals. J.D.L. is a paid Advisory Board member for F. Hoffmann–La Roche and uniQure biopharma and is a paid consultant for Vaccinex, Wave Life Sciences USA, Genentech, Triplet and PTC Therapeutics. T.H.M. is an associate member of the scientific advisory board of LoQus23 Therapeutics and has consulted for Transine Therapeutics (dba Harness Therapeutics). P.H. is a member of the Enroll-HD Scientific Review Committee. S.K. and C.S. are employed by CHDI Management as advisors to the CHDI Foundation. E.R.D. has provided consulting services to Abbott, Abbvie, Acadia, Acorda Therapeutics, Biogen, Biohaven Pharmaceuticals, BioSensics, Boehringer Ingelheim, Caraway Therapeutics, Cerevance, CuraSen, DConsult2, Denali Therapeutics, Eli Lilly, Genentech, Health & Wellness Partners, HMP Education, Karger, KOL groups, Life Sciences Consultant, Mediflix, Medrhythms, Merck, Mitsubishi Tanabe Pharma America, MJH Holdings, NACCME Novartis, Otsuka, Praxis Medicine, Sanofi, Seelos Therapeutics, Spark Therapeutics, Springer Healthcare, Theravance Biopharmaceuticals and WebMD. He has received grant support from Averitas Pharma, Biogen, Burroughs Wellcome Fund, Pfizer, Photopharmics and Roche and has an ownership interest in Included Health, Mediflix, SemCap and Synapticure. G.B.L. has provided consulting services, scientific advice, scientific advisory board functions, independent data monitoring committee services and/or lectures for Acadia Pharmaceuticals, Affiris, Allergan, Alnylam, Amarin, AOP Orphan Pharmaceuticals, Bayer Pharma, Boehringer Ingelheim, CHDI Foundation, Deutsche Huntington-Hilfe, Desitin, Genentech, Genzyme, GlaxoSmithKline, F. Hoffmann–La Roche, Ipsen, ISIS Pharma (IONIS), Lilly, Lundbeck, Medesis, Medivation, Medtronic, NeuraMetrix, Neurosearch, Novartis, Pfizer, Prana Biotechnology, Prilenia, PTC Therapeutics, Raptor, Remix Therapeutics, Rhône-Poulenc Rorer, Roche Pharma AG Deutschland, Sage Therapeutics, Sanofi-Aventis, Sangamo/Shire, Siena Biotech, Takeda, Temmler Pharma GmbH, Teva, Triplet Therapeutics, Trophos, UniQure and Wave Life Sciences. R.H.M. is a paid advisory board member for Rgenta Therapeutics and is on the scientific advisory board with financial interests in Gatehouse Bio. All other authors declare no competing interests.
