Genotyping single nucleotide polymorphisms for allele-selective therapy in Huntington disease
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Article Information
- Received September 17, 2019
- Accepted in final form February 24, 2020
- First Published May 14, 2020.
Author Disclosures
- Daniel O. Claassen, MD,
- Jody Corey-Bloom, MD, PhD,
- E. Ray Dorsey, MD,
- Mary Edmondson, MD,
- Sandra K. Kostyk, MD, PhD,
- Mark S. LeDoux, MD, PhD,
- Ralf Reilmann, MD,
- H. Diana Rosas, MD,
- Francis Walker, MD,
- Vicki Wheelock, MD,
- Nenad Svrzikapa, MS,
- Kenneth A. Longo, PhD,
- Jaya Goyal, PhD,
- Serena Hung, MD and
- Michael A. Panzara, MD, MPH
- Daniel O. Claassen, MD,
Teva Neuroscience, Wave Life Sciences
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Medscape, honorarium Southern Clinical Neuroscience, travel reimbursement Rockpoint, honoriarum
HD Insights, Editor, 2019-present Mayo Clinic Proceedings, Associate Editor, 2019-present
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Acadia, Adamas, Teva Neuroscience, Vertical Pharmaceuticals
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AbbVie, Acadia, Biogen, BMS, Genetech/Roche, Wave Life Sciences, Vaccinex
NIH/NINDS, R01 NS097783, PI, 2016-2021 NIH/NINDS, K23 NS080988, PI, 2013-2019 Department of Defense, W81XWH-19-1-0812, PI, 2019-2022
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CHDI Inc., HDSA, Michael J Fox Foundation
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- Jody Corey-Bloom, MD, PhD,
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Speaker honoraria from Teva Pharmaceuticals and Genentech.
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Teva Pharmaceuticals and Genentech
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Clinical trial support from Genentech, Vaccinex, Wave Pharmaceuticals, and CHDI.
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- E. Ray Dorsey, MD,
Medical Advisory Board, Grand Rounds, an online second opinion service
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received travel funding and/or honoraria from the American Academy of Neurology, American Neurological Association, University of Michigan
Editorial board member for the Journal of Huntington's Disease; Editor of HD Insights; Editor of Digital Biomarkers
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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,
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Clinical practice, including telemedicine, as a movement disorder neurologist (20%)
Abbvie, AMC Health, Avid Radiopharmaceuticals, BioMarin, GlaxoSmithKline, Great Lakes Neurotechnologies, Lundbeck, Medtronic, Prana Biotechnology, Raptor Pharmaceuticals, Roche, Teva Pharmaceuticals
National Institute of Neurological Disorders and Stroke, Patient-Centered Outcomes Research Institute
University of California Irvine, Duke University
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)
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has received compensation for expert testimony
- Mary Edmondson, MD,
1. Wave life Sciences 2. uniQure N.V.
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Huntington Study Group
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- Sandra K. Kostyk, MD, PhD,
Lundbeck Advisory Board honorarium and travel costs Prilenia Advisory Board honorarium and travel costs
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NIH, honorarium and travel costs Wave Life Sciences travel costs Huntington's Disease Society of America travel costs Huntington Study Group travel costs
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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
NIH. NeuroNext STAIR Study 105, Site PI: Kostyk and Kissel. 2 years.
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Craig Neilson Foundation CHDI Foundation
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- Mark S. LeDoux, MD, PhD,
(1) Chairman, Board of Medical Advisors, National Spasmodic Torticollis Association (2) Member, Scientific Advisory Board, Spastic Paraplegia Foundation
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(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
Tremor and Other Hyperkinetic Movement Disorders, Editorial Board, 2011-present. Neurology, Editorial Board, 2015-present.
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Elsevier, Animal Models of Movement Disorders, 2005- present. Elsevier, Movement Disorders: Genetics and Models, 2015- present TheBookPatch, Parkinson's Disease Poetry, 2018
(1) University of Memphis, 2018 to present (2) Veracity Neuroscience LLC, 2019 to present
US WorldMeds
(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
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(1) Michael J. Fox Foundation, ÂA 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 ParkinsonÂs Disease (PD)(NILO-PD)Â(LeDoux - Site PI), 12JAN2018- present 3 (3) Axovant Sciences, Inc., Axovant 2003, Study REM SBD 2003 Â 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 ParkinsonÂs Disease Dementia (PDD) who have REM sleep behavior disorder (RBD) (LeDoux - Site PI),20AUG2017 Â present (5)Wave Life Sciences, Wave Observational Study, Study 17- 0005-CL-P-02 - TV5600-CNS-20007 Â Observational Study to Determine the Presence of Single Nucleotide Polymorphisms rs362307(T/C) and rs362331(T/C) in Patients with HuntingtonÂs Disease (LeDoux - Site PI), 25APR2017 Â 30JUN2018 (6)Teva, LEGATO, Study TV5600-CNS-20007 Â 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 Â 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
(1) National Institutes of Health, Dystonia Coalition, Prospective Natural History Study and Biorepository, 2011 to present (2) DOD (LeDoux-PI), ÂDNA Repair in Dystonia, 2017-2018 (3) NIH 1R56NS094965 (LeDoux-PI), ÂDefining 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), ÂMouse 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, ÂStudy of Urate Elevation in Parkinson's Disease, Phase 3 (SURE- PD3) 2016-2019
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Benign Essential Blepharospasm Research Foundation Dystonia Medical Research Foundation
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- Ralf Reilmann, MD,
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
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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
Journal of Huntington Disease (associate editor)
Patents for Q-Motor quantitative motor assessments
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Founding Director, C.E.O. and owner of George-Huntington-Institut GmbH (GHI) and QuantiMedis GmbH, Muenster, Germany
(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
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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)
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Provided to GHI/QM: (1) Pfizer, (2) Teva, (3) Vaccinex, (4) uniQure
(1) EHDN, (2) EU-FP-7 Repair-HD, (3) IMI2 IDEA-FAST, (4) NIH CHILD-HD
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(1) CHDI Foundation
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- H. Diana Rosas, MD,
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1) Vaccinex, Inc 2) Voyager
NIH NINDS P01 NS058793 NIH, NINDS R01 NS042861 NIH, NCCAM U01 AT000613
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- Francis Walker, MD,
I am on the data safety monitoring committee for a study of intrathecal oxytocin which is being conducted by a non- profit entity.
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The International Society of Peripheral Neurophysiologic Imaging, travel and speaker honoraria, and Huntington Study Group, travel.
Muscle and Nerve: Editorial board and Associate Editor (2011-2020) Journal of Clinical Neurophysiology: Editorial Board: (2014-2020)
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1) Neuromuscular Ultrasound (Textbook): Elsevier, 2011 2) Diagnostic ultrasound in neuromuscular disease: Up to Date 2011
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I have interest free ultrasound equipment loans from Terason, Natus, and Monarch Medical
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Commercial entity: Vaccinex, Wave Life Sciences
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CHDI: ENROLL-HD an observational study of Huntington's disease. CLARITY-HD an observational biomarker study, part of ENROLL-HD.
Chaperone therapeutics.
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Chaperone Therapeutics
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- Vicki Wheelock, MD,
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(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
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(1)Genentech, HD Education Day (2)Hoffman-La Roche Pharmaceuticals, Advisory Board meeting (3)uniQure, AMT-130 Steering Committee Member
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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
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(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
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- Nenad Svrzikapa, MS,
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I am employed by Wave Life Sciences.
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I was/am employed by Wave Life Sciences.
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Employee compensation by Wave Life Sciences, including stocks which are given on yearly basis to all employees.
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- Kenneth A. Longo, PhD,
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I am employed by WAVE Life Science Ltd as Sr. Director of Data Science. I've been at WAVE since August 2016.
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I receive a salary from WAVE Life Sciences Ltd.
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- Jaya Goyal, PhD,
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Wave Life Sciences Vice President 2.11 years
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- Serena Hung, MD and
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Wave Life Sciences, Senior Medical Director, 2.5 years
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I am a full time employee of Wave Life Sciences
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I have stocks and stock options in Wave Life Sciences 2017- present
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- Michael A. Panzara, MD, MPH
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(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
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- 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.
- Correspondence
Dr. Claassen Daniel.Claassen{at}vumc.org
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