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October 22, 2019; 93 (17) Article

Relationships between DMD mutations and neurodevelopment in dystrophinopathy

Mathula Thangarajh, Jos Hendriksen, Michael P. McDermott, William Martens, Kimberly A. Hart, Robert C. Griggs, for The Muscle Study Group and TREAT-NMD
First published October 8, 2019, DOI: https://doi.org/10.1212/WNL.0000000000008363
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Article Information

vol. 93 no. 17 e1597-e1604
DOI: 
https://doi.org/10.1212/WNL.0000000000008363
PubMed: 
31594858
Published By: 
Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology
Print ISSN: 
0028-3878
Online ISSN: 
1526-632X
History: 
  • Received December 20, 2018
  • Accepted in final form May 22, 2019
  • First Published October 8, 2019.

Article Versions

Copyright & Usage: 
© 2019 American Academy of Neurology

Author Disclosures

    1. Mathula Thangarajh, MD, PhD,
    2. Jos Hendriksen, PhD,
    3. Michael P. McDermott, PhD,
    4. William Martens, BA,
    5. Kimberly A. Hart, MA,
    6. Robert C. Griggs, MD;
    7. for The Muscle Study Group and TREAT-NMD
  1. Mathula Thangarajh, MD, PhD,
    2. Scientific Advisory Boards:

    1. PTC, Biogen

    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. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. Mathula Thangarajh was receipent of the American Academy of Neurology Clinical Research Training Fellowship from 2015 to 2017.

    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

  2. Jos Hendriksen, PhD,
    3. 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. 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

  3. Michael P. McDermott, PhD,
    4. Scientific Advisory Boards:

    1. AstraZeneca and Eli Lilly and Company (DSMB for clinical trials in Alzheimer's disease), aTyr Pharma (DSMB for a clinical trial in facioscapulohumeral muscular dystrophy), NIH (DSMB for a clinical trial in the elderly following hip fracture); Massachusetts General Hospital (DSMB for a clinical trial in hereditary sensory and autonomic neuropathy Type I); Catabasis Pharmaceuticals, Inc. (DSMB for clinical trials in Duchenne muscular dystrophy); Vaccinex, Inc. (DSMB for a clinical trial in Huntington's disease); Cynapsus Therapeutics (DSMB for clinical trials in Parkinson's disease); Voyager Therapeutics (DSMB for a clinical trial in Parkinson's disease).

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. Movement Disorders, Editorial Board, 2010-2019; Chance, Editor, 2014-2019.

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. Received consulting fees from Voyager Therapeutics (2017).

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. Received grant support from PTC Therapeutics.

    Research Support, Government Entities:

    1. Received grant support from: NIH, NS048843, Co-I, 2003- present; NIH, NS061795, PI, 2010-present; NIH, NS101944, Co- I, 2017-present; NIH, NS095813, Co-I, 2017-present; NIH, NS102176, Co-I, 2017-present; NIH, NS065712, Co-I, 2009- present; NIH, AR065119, Co-I, 2013-2017; NIH, EY025990, Co- I, 2017-present; NIH, NS080840, Co-I, 2014-present; FDA, FD003716, Co-I, 2011-2017; FDA, FD005936, Co-I, 2016- present; FDA, FD004809, Co-I, 2015-present; NYSTEM, C028502, Co-I, 2013-2018; NIH, AR068052, PI, 2016-2018; NIH, NS108676, Co-I, 2018-present.

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. Received grant support from the Spinal Muscular Atrophy Foundation, Cure SMA, and the Parkinson Study Group.

    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

  4. William Martens, BA,
    5. 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. 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. Kimberly A. Hart, MA,
    6. 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. NONE

    Research Support, Government Entities:

    1. NIH/NINDS,NS061799, FOR-DMD Project Manager, 2010-2019

    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

  6. Robert C. Griggs, MD;
    7. Scientific Advisory Boards:

    1. 1. DSMB-Solid Bioscience 2. DSMB-Idera Pharmaceuticals

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Strongbridge BioPharma-Speaker commercial

    Editorial Boards:

    1. NeuroTherapeutics. Editorial Board No compensation Correspondence Editor, Neurology. annual stipend. (ended 2018)

    Patents:

    1. NONE

    Publishing Royalties:

    1. Andreoli and Carpenter’s Cecil Essentials of Medicine, Eighth Edition. Elsevier, 2000, 2004, 2007 and 2010, 2015 Cecil Textbook of Medicine, multiple editions, Elsevier, 2000, 2004, 2008, 2010 and 2011, 2015, 2018 Eavulation and Treatment of Myopathies, Oxford, 2014

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. Strongbridge BioPharma and Stealth BioPharma

    Speakers' Bureaus:

    1. Strongbridge BioPharma

    Other activities:

    1. Royalties for data of previous studies from Taro Pharmaceuticals and PTC Pharmaceuticals. Supported by PTC and Sarepta to supplement current NIH grant

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. T32 NS07338 Griggs (PI and preceptor, 7/1/15-6/30/20, NINDS, Experimental Therapeutics of Neurological Disease Griggs (PI), 3/1/10-2/28/16, NINDS 5U01NS061799, FOR-DMD: Double-blind randomized trial to optimize steroid regimen in Duchenne MD Griggs, Site-PI, 2/15/11-1/31/15, FDA R01 FD003923 (Jen PI) A Randomized Trial on the Efficacy and Tolerability of 4-Aminopyridine in EA2 Griggs (PI), 8/1/13-7/31/14, NINDS 1R13NS084687, Novel Molecular Mechanisms of Neuromuscular Disease: Implications for Therapy.

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. Parent Project Muscular Dystrophy, Inc., Griggs (PI), 1/1/2013-1/1/2016, Predicting the Benefits Vs. the Side Effects of Corticosteroids in Duchenne Muscular Dystrophy Muscular Dystrophy Association, Griggs (PI),10/1/2013- 9/20/2016, FOR-DMD:Double-Blind Randomized Trial to Optimize Steroid Regimen in Duchenne MD Muscular Dystrophy Association,Griggs (PI), 7/1/2013 – 6/30/2014, 287108, Novel Molecular Mechanisms of Neuromuscular Disease: Implications for Therapy

    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. for The Muscle Study Group and TREAT-NMD
  1. From the Department of Neurology (M.T.), Children's National Health System, Washington, DC; Kempenhaeghe Center for Neurological Learning Disabilities (J.H.), Heeze, the Netherlands; and School of Medicine and Dentistry (M.P.M., W.M., K.A.H., R.C.G.), University of Rochester Medical Center, NY. M.T. is currently affiliated with the Department of Neurology, Virginia Commonwealth University, Richmond.
  1. Correspondence
    Dr. Thangarajh mathula.thangarajh{at}vcuhealth.org
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