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June 2020; 6 (3) ArticleOpen Access

Genotyping single nucleotide polymorphisms for allele-selective therapy in Huntington disease

Daniel O. Claassen, Jody Corey-Bloom, E. Ray Dorsey, Mary Edmondson, Sandra K. Kostyk, Mark S. LeDoux, Ralf Reilmann, H. Diana Rosas, Francis Walker, Vicki Wheelock, Nenad Svrzikapa, Kenneth A. Longo, Jaya Goyal, Serena Hung, Michael A. Panzara
First published May 14, 2020, DOI: https://doi.org/10.1212/NXG.0000000000000430
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Article Information

vol. 6 no. 3 e430
DOI: 
https://doi.org/10.1212/NXG.0000000000000430
Published By: 
Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology
Online ISSN: 
2376-7839
History: 
  • Received September 17, 2019
  • Accepted in final form February 24, 2020
  • First Published May 14, 2020.
Copyright & Usage: 
Copyright © 2020 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology. This is an open access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives License 4.0 (CC BY-NC-ND), which permits downloading and sharing the work provided it is properly cited. The work cannot be changed in any way or used commercially without permission from the journal.

Author Disclosures

    1. Daniel O. Claassen, MD,
    2. Jody Corey-Bloom, MD, PhD,
    3. E. Ray Dorsey, MD,
    4. Mary Edmondson, MD,
    5. Sandra K. Kostyk, MD, PhD,
    6. Mark S. LeDoux, MD, PhD,
    7. Ralf Reilmann, MD,
    8. H. Diana Rosas, MD,
    9. Francis Walker, MD,
    10. Vicki Wheelock, MD,
    11. Nenad Svrzikapa, MS,
    12. Kenneth A. Longo, PhD,
    13. Jaya Goyal, PhD,
    14. Serena Hung, MD and
    15. Michael A. Panzara, MD, MPH
  1. Daniel O. Claassen, MD,
    2. Scientific Advisory Boards:

    1. Teva Neuroscience, Wave Life Sciences

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Medscape, honorarium Southern Clinical Neuroscience, travel reimbursement Rockpoint, honoriarum

    Editorial Boards:

    1. HD Insights, Editor, 2019-present Mayo Clinic Proceedings, Associate Editor, 2019-present

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. Acadia, Adamas, Teva Neuroscience, Vertical Pharmaceuticals

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. AbbVie, Acadia, Biogen, BMS, Genetech/Roche, Wave Life Sciences, Vaccinex

    Research Support, Government Entities:

    1. NIH/NINDS, R01 NS097783, PI, 2016-2021 NIH/NINDS, K23 NS080988, PI, 2013-2019 Department of Defense, W81XWH-19-1-0812, PI, 2019-2022

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. CHDI Inc., HDSA, Michael J Fox Foundation

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  2. Jody Corey-Bloom, MD, PhD,
    3. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Speaker honoraria from Teva Pharmaceuticals and Genentech.

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. Teva Pharmaceuticals and Genentech

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. Clinical trial support from Genentech, Vaccinex, Wave Pharmaceuticals, and CHDI.

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  3. E. Ray Dorsey, MD,
    4. Scientific Advisory Boards:

    1. Medical Advisory Board, Grand Rounds, an online second opinion service

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. received travel funding and/or honoraria from the American Academy of Neurology, American Neurological Association, University of Michigan

    Editorial Boards:

    1. Editorial board member for the Journal of Huntington's Disease; Editor of HD Insights; Editor of Digital Biomarkers

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. 23andMe, Abbott, Abbvie, American Well Biogen, Clintrex, DeciBio, Denali Therapeutics, GlaxoSmithKline, Grand Rounds, Karger, Lundbeck, MC10, MedAvante, Medico Legal services, Mednick Associates, NIH/NINDS, Olson Research Group, Optio, Origent Data Sciences, Prilenia, Putnam Associates, Roche, Sanofi, Shire, Sunovion Pharma, Teva, UCB, Voyager Therapeutics,

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical procedures or imaging studies:

    1. Clinical practice, including telemedicine, as a movement disorder neurologist (20%)

    Research Support, Commercial Entities:

    1. Abbvie, AMC Health, Avid Radiopharmaceuticals, BioMarin, GlaxoSmithKline, Great Lakes Neurotechnologies, Lundbeck, Medtronic, Prana Biotechnology, Raptor Pharmaceuticals, Roche, Teva Pharmaceuticals

    Research Support, Government Entities:

    1. National Institute of Neurological Disorders and Stroke, Patient-Centered Outcomes Research Institute

    Research Support, Academic Entities:

    1. University of California Irvine, Duke University

    Research Support, Foundations and Societies:

    1. Burroughs Wellcome Fund, Davis Phinney Foundation, Michael J. Fox Foundation, Safra Foundation, Greater Rochester Health Foundation, Huntington Study Group, National Science Foundation Stock/Stock Options, Medical Equipment & Materials: Grand Rounds (stock options) BlackFynn (stock options)

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. has received compensation for expert testimony

  4. Mary Edmondson, MD,
    5. Scientific Advisory Boards:

    1. 1. Wave life Sciences 2. uniQure N.V.

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Huntington Study Group

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  5. Sandra K. Kostyk, MD, PhD,
    6. Scientific Advisory Boards:

    1. Lundbeck Advisory Board honorarium and travel costs Prilenia Advisory Board honorarium and travel costs

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. NIH, honorarium and travel costs Wave Life Sciences travel costs Huntington's Disease Society of America travel costs Huntington Study Group travel costs

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. Vaccinex: support for participation in multicenter clinical trial Voyager Therapeutics: support for participation in multicenter clinical trial uniQure: support for participation in multicenter clinical trial Wave LifeScience: support for participation in multicenter clinical trial

    Research Support, Government Entities:

    1. NIH. NeuroNext STAIR Study 105, Site PI: Kostyk and Kissel. 2 years.

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. Craig Neilson Foundation CHDI Foundation

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  6. Mark S. LeDoux, MD, PhD,
    7. Scientific Advisory Boards:

    1. (1) Chairman, Board of Medical Advisors, National Spasmodic Torticollis Association (2) Member, Scientific Advisory Board, Spastic Paraplegia Foundation

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. (1)Adamas Pharmaceuticals, Speaker (2)Acadia Pharmaceuticals, Speaker (3)Teva Pharmaceutical Industries, Speaker (4)US WorldMeds, Speaker (5)Kyowa Kirin, Speaker (6)Acorda Therapeutics, Speaker

    Editorial Boards:

    1. Tremor and Other Hyperkinetic Movement Disorders, Editorial Board, 2011-present. Neurology, Editorial Board, 2015-present.

    Patents:

    1. NONE

    Publishing Royalties:

    1. Elsevier, Animal Models of Movement Disorders, 2005- present. Elsevier, Movement Disorders: Genetics and Models, 2015- present TheBookPatch, Parkinson's Disease Poetry, 2018

    Employment, Commercial Entity:

    1. (1) University of Memphis, 2018 to present (2) Veracity Neuroscience LLC, 2019 to present

    Consultancies:

    1. US WorldMeds

    Speakers' Bureaus:

    1. (1)Adamas Pharmaceuticals, Speaker (2)Acadia Pharmaceuticals, Speaker (3)Teva Pharmaceutical Industries, Speaker (4)US WorldMeds, Speaker (5)Kyowa Kirin, Speaker (6)Acorda Therapeutics, Speaker

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. (1) Michael J. Fox Foundation, “A Randomized, Double-Blind, Placebo-Controlled, Phase IIa, Parallel Group, Two-Cohort Study to Define the Safety, Tolerability, Clinical and Exploratory Biological Activity of the Chronic Administration of Nilotinib in Participants with Parkinson’s Disease (PD)(NILO-PD)”(LeDoux - Site PI), 12JAN2018- present 3 (3) Axovant Sciences, Inc., Axovant 2003, Study REM SBD 2003 – An open-label study of nelotanserin in patients with Lewy body dementia who have frequent visual hallucinations or REM sleep behaviors (LeDoux - Site PI), 27SEP2017 - present (4) Axovant Sciences, Inc., Axovant 2002, Study RVT-102- 2002 - A Phase 2, double-blind, randomized, placebo- controlled study of nelotanserin versus placebo in patients with dementia with Lewy bodies (DLB) or Parkinson’s Disease Dementia (PDD) who have REM sleep behavior disorder (RBD) (LeDoux - Site PI),20AUG2017 – present (5)Wave Life Sciences, Wave Observational Study, Study 17- 0005-CL-P-02 - TV5600-CNS-20007 – Observational Study to Determine the Presence of Single Nucleotide Polymorphisms rs362307(T/C) and rs362331(T/C) in Patients with Huntington’s Disease (LeDoux - Site PI), 25APR2017 – 30JUN2018 (6)Teva, LEGATO, Study TV5600-CNS-20007 – A Multicenter, Multinational, Randomized, Double-Blind, Placebo- Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Laquinimod (0.5, 1.0 and 1.5 mg/day) as Treatment in Patients with Huntington's Disease (LeDoux - Site PI), 01MAR2017 – present (7) Pharma Two B Ltd., A Phase 3, Twelve-week Study to Determine the Efficacy, Safety and Tolerability of P2B001 Once Daily Compared to Its Individual Components in Subjects with Early Parkinson's Disease and to a Calibration Arm of Pramipexole ER (LeDoux - Site PI), 01OCT2018-19JUL2019

    Research Support, Government Entities:

    1. (1) National Institutes of Health, Dystonia Coalition, Prospective Natural History Study and Biorepository, 2011 to present (2) DOD (LeDoux-PI), “DNA Repair in Dystonia,” 2017-2018 (3) NIH 1R56NS094965 (LeDoux-PI), “Defining the neuroanatomical substrates of dystonia using mouse models of paroxysmal kinesigenic dyskinesia (PKD) and identifying the in vivo and in vitro neurophysiological abnormalities that drive dystonia,” 2017-2018 (4)NIH R03 NS101485-01A1 (LeDoux-PI), “Mouse Models of Paroxysmal Non-Kinesigenic Dyskinesia,” 2018 (5)NIH/NINDS, R01NS082296, Genetics and Biology of CIZ1 in Cervical Dystonia, 2013-2018 (6) R21 GM118962, The Role of UBTF in Undiagnosed Neurodevelopmental Disorders, NIH(Hori and LeDoux, Co- PIs) 2016-2019 (7)5U01NS090259 (LeDoux - Site PI), NIH/NINDS, “Study of Urate Elevation in Parkinson's Disease, Phase 3 (SURE- PD3)” 2016-2019

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. Benign Essential Blepharospasm Research Foundation Dystonia Medical Research Foundation

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  7. Ralf Reilmann, MD,
    8. Scientific Advisory Boards:

    1. All compensation provided to GHI: (1) Alnylam, (2) AOP Orphan, (3) CHDI, (4) Desitin, (5) Eli Lilly, (6) Ionis, (7) Ipsen, (8) Lundbeck, (9) Mitoconix, (10) Neurocrine, (11) Novartis, (12) Pfizer, (13) Prilenia, (14) Roche, (15) Spark, (16) Takeda, (17) TEVA, (18) Triplet Therapeutics, (19) WAVE, (20) UBC, (21) uniQure, (22) Vaccinex, (23) Voyager

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. All compensation provided to GHI: (1) Alnylam, (2) AOP Orphan, (3) CHDI, (4) Desitin, (5) Eli Lilly, (6) Ionis, (7) Ipsen, (8) Lundbeck, (9) Mitoconix, (10) Neurocrine, (11) Novartis, (12) Pfizer, (13) Prilenia, (14) Roche, (15) Spark, (16) Takeda, (17) TEVA, (18) Triplet Therapeutics, (19) WAVE, (20) UBC, (21) uniQure, (22) Vaccinex, (23) Voyager

    Editorial Boards:

    1. Journal of Huntington Disease (associate editor)

    Patents:

    1. Patents for Q-Motor quantitative motor assessments

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. Founding Director, C.E.O. and owner of George-Huntington-Institut GmbH (GHI) and QuantiMedis GmbH, Muenster, Germany

    Consultancies:

    1. (1) Alnylam, (2) AOP Orphan, (3) CHDI, (4) Desitin, (5) Eli Lilly, (6) Ionis, (7) Ipsen, (8) Lundbeck, (9) Mitoconix, (10) Neurocrine, (11) Novartis, (12) Pfizer, (13) Prilenia, (14) Roche, (15) Spark, (16) Takeda, (17) TEVA, (18) Triplet Therapeutics, (19) WAVE, (20) UBC, (21) uniQure, (22) Vaccinex, (23) Voyager

    Speakers' Bureaus:

    1. NONE

    Other activities:

    1. Co-Chair of MDS Task Force on Huntington Disease, Member MDS Task Force on Technology, Member Executive Committee European HD Network (EHDN), Member Executive Committee Huntington Study Group (HSG)

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. Provided to GHI/QM: (1) Pfizer, (2) Teva, (3) Vaccinex, (4) uniQure

    Research Support, Government Entities:

    1. (1) EHDN, (2) EU-FP-7 Repair-HD, (3) IMI2 IDEA-FAST, (4) NIH CHILD-HD

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. (1) CHDI Foundation

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  8. H. Diana Rosas, MD,
    9. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. 1) Vaccinex, Inc 2) Voyager

    Research Support, Government Entities:

    1. NIH NINDS P01 NS058793 NIH, NINDS R01 NS042861 NIH, NCCAM U01 AT000613

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  9. Francis Walker, MD,
    10. Scientific Advisory Boards:

    1. I am on the data safety monitoring committee for a study of intrathecal oxytocin which is being conducted by a non- profit entity.

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. The International Society of Peripheral Neurophysiologic Imaging, travel and speaker honoraria, and Huntington Study Group, travel.

    Editorial Boards:

    1. Muscle and Nerve: Editorial board and Associate Editor (2011-2020) Journal of Clinical Neurophysiology: Editorial Board: (2014-2020)

    Patents:

    1. NONE

    Publishing Royalties:

    1. 1) Neuromuscular Ultrasound (Textbook): Elsevier, 2011 2) Diagnostic ultrasound in neuromuscular disease: Up to Date 2011

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other activities:

    1. I have interest free ultrasound equipment loans from Terason, Natus, and Monarch Medical

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. Commercial entity: Vaccinex, Wave Life Sciences

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. CHDI: ENROLL-HD an observational study of Huntington's disease. CLARITY-HD an observational biomarker study, part of ENROLL-HD.

    Stock/Stock Options/Board of Directors Compensation:

    1. Chaperone therapeutics.

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. Chaperone Therapeutics

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  10. Vicki Wheelock, MD,
    11. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. (1) Hoffman-La Roche, Advisory Board meeting member (2) uniQure, AMT-130 Steering Committee member meeting (3) Huntington's Disease Society of America, annual convention speaker

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. (1)Genentech, HD Education Day (2)Hoffman-La Roche Pharmaceuticals, Advisory Board meeting (3)uniQure, AMT-130 Steering Committee Member

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. 1. Huntington Study Group. Role: Steering Committee Member, SIGNAL Trial 2. Wave Life Sciences, site investigator 2. Genentech, site investigator 3. uniQure, steering committee co-Chair

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. (1)CHDI, sponsor for Enroll-HD Study; site investigator (2)Huntington's Disease Society of America, Center of Excellence Grant (3)Joseph P. Roberson Foundation, UC Davis Huntington's Disease Clinic Support

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  11. Nenad Svrzikapa, MS,
    12. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. I am employed by Wave Life Sciences.

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. I was/am employed by Wave Life Sciences.

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. Employee compensation by Wave Life Sciences, including stocks which are given on yearly basis to all employees.

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  12. Kenneth A. Longo, PhD,
    13. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. I am employed by WAVE Life Science Ltd as Sr. Director of Data Science. I've been at WAVE since August 2016.

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. I receive a salary from WAVE Life Sciences Ltd.

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  13. Jaya Goyal, PhD,
    14. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. Wave Life Sciences Vice President 2.11 years

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  14. Serena Hung, MD and
    15. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. Wave Life Sciences, Senior Medical Director, 2.5 years

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. I am a full time employee of Wave Life Sciences

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. I have stocks and stock options in Wave Life Sciences 2017- present

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  15. Michael A. Panzara, MD, MPH
    16. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. (1) Wave Life Sciences, Chief Medical Officer, 3.5 years (2) Sanofi Genzyme, Head, Multiple Sclerosis, Neurology, Ophthalmology Therapeutic Area, 8 years Stock/Stock Options, Medical Equipment & Materials: (1) Wave Life Sciences, Chief Medical Officer, 3.5 years (2) Sanofi Genzyme, Head, Multiple Sclerosis, Neurology, Ophthalmology Therapeutic Area, 8 years

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  1. From the Vanderbilt University Medical Center (D.O.C.), Nashville, TN; University of California San Diego (J.C.-B.), La Jolla; University of Rochester Medical Center (E.R.D.), NY; HD Reach (M.E.), Raleigh, NC; Ohio State University (S.K.K.), Columbus; University of Memphis and Veracity Neuroscience, LLC (M.S.L.), TN; George-Huntingon-Institute & Department of Clinical Radiology University of Muenster (R.R.), Department of Neurodegeneration, Hertie Institute for Clinical Brain Research, University of Tuebingen, Germany; Havard Medical School (H.D.R.), Massachusetts General Hospital, Boston; Wake Forest University School of Medicine (F.W.), Winston Salem, NC; University of California Davis Health (V.W.), Sacramento, CA; Wave Life Sciences USA, Inc. (N.S., K.A.L., J.G., S.H., M.A.P.), Cambridge, MA; and Department of Paediatrics (N.S.), Medical Sciences Division, University of Oxford, UK.
  1. Correspondence
    Dr. Claassen Daniel.Claassen{at}vumc.org
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