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October 26, 2021; 97 (17) Research Article

Association Between Body Mass Index and Disability in Children With Charcot-Marie-Tooth Disease

Gabrielle A. Donlevy, Sarah P. Garnett, Kayla M.D. Cornett, Marnee J. McKay, Jennifer N. Baldwin, Rosemary R. Shy, Sabrina W. Yum, Timothy Estilow, Isabella Moroni, Maria Foscan, Emanuela Pagliano, Davide Pareyson, Matilde Laura, Trupti Bhandari, Francesco Muntoni, Mary M. Reilly, Richard S. Finkel, Janet E. Sowden, Katy J. Eichinger, David N. Herrmann, Michael E. Shy, Joshua Burns, Manoj P. Menezes
First published September 7, 2021, DOI: https://doi.org/10.1212/WNL.0000000000012725
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Article Information

vol. 97 no. 17 e1727-e1736
DOI: 
https://doi.org/10.1212/WNL.0000000000012725
PubMed: 
34493614
Published By: 
Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology
Print ISSN: 
0028-3878
Online ISSN: 
1526-632X
History: 
  • Received February 15, 2021
  • Accepted in final form August 16, 2021
  • First Published September 7, 2021.

Article Versions

Copyright & Usage: 
© 2021 American Academy of Neurology

Author Disclosures

    1. Gabrielle A. Donlevy, MND,
    2. Sarah P. Garnett, PhD,
    3. Kayla M.D. Cornett, PhD,
    4. Marnee J. McKay, PhD,
    5. Jennifer N. Baldwin, PhD,
    6. Rosemary R. Shy, MD,
    7. Sabrina W. Yum, MD,
    8. Timothy Estilow, OTR/L,
    9. Isabella Moroni, MD,
    10. Maria Foscan, PT,
    11. Emanuela Pagliano, MD,
    12. Davide Pareyson, MD,
    13. Matilde Laura, MD, PhD,
    14. Trupti Bhandari, PT,
    15. Francesco Muntoni, MD, FRCPCH, FMed,
    16. Mary M. Reilly, MD, FRCP,
    17. Richard S. Finkel, MD,
    18. Janet E. Sowden, BSc,
    19. Katy J. Eichinger, PhD,
    20. David N. Herrmann, MBBCh,
    21. Michael E. Shy, MD,
    22. Joshua Burns, PhD* and
    23. Manoj P. Menezes, MD, PhD*
  1. Gabrielle A. Donlevy, MND,
    2. 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. (1) GA Donlevy acknowledges support from The University of Sydney, Postgraduate Research Scholarship in Childhood Inherited Neuropathy

    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. Sarah P. Garnett, PhD,
    3. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. Clinical Obesity (The World Obesity Federation) Deputy Editor 2019 ongoing

    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. 2017-2021 National Health and Medical Research Council (NHMRC)/Project Grants CI The alternate day fasting diet in adolescents with obesity: a randomised controlled trial Fast Track Trial

    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. Kayla M.D. Cornett, PhD,
    4. 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

  4. Marnee J. McKay, PhD,
    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. Centre for Research Excellence in Neuromuscular Disorders National Health and Medical Research Council of Australia 2013-2016(NHMRC #1031893)

    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. Jennifer N. Baldwin, PhD,
    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. (1) Auckland University of Technology

    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

  6. Rosemary R. Shy, MD,
    7. 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

  7. Sabrina W. Yum, MD,
    8. Scientific Advisory Boards:

    1. Commercial entities: PTC Therapeutics, Inc Sarepta Therapeutics

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. PTC Therapeutics, Inc Sarepta Therapeutics

    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. Muscular Dystrophy Association (through the INC Consortium)

    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. Timothy Estilow, OTR/L,
    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. 1) Commercial (Sarepta Therapeutics) Speakers Bureau Educational Presentations on DMD Honorarium and travel reimbursement

    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

  9. Isabella Moroni, MD,
    10. 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

  10. Maria Foscan, PT,
    11. 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

  11. Emanuela Pagliano, MD,
    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. 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

  12. Davide Pareyson, MD,
    13. Scientific Advisory Boards:

    1. 1) Commercial entity: Member of the Scientific Advisory Board of Inflectis 2) Commercial entity: participated in Scientific Advisory Meetings of Alnylam 3) Commercial entity: participated in Scientific Advisory Meetings of Akcea

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. 1) Commercial entity: funding for Travel and Honorarium for speaking at a Symposium organized by Alnylam in July 2019

    Editorial Boards:

    1. Neurological Sciences, member of the advisory board (2006-....) Journal of Neuromuscular Disorders, member of the editorial board (2014-....) Neurology Genetics, member of the editorial board (2017-....)

    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. 1) Telethon Italy (GUP15009; GUP 15010; GPP19099) 2) AFM-Telethon (#20667; #20821) 3) Charcot-Marie-Tooth Association (CMTA) 4) NIH grant (Subaward # 417583/UR FAO GR510930)

    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. Matilde Laura, MD, 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. 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

  14. Trupti Bhandari, PT,
    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. 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

  15. Francesco Muntoni, MD, FRCPCH, FMed,
    16. Scientific Advisory Boards:

    1. (1) Pfizer, Scientific Advisory Board (2) Sarepta, Scientific advisory board (3) PTC Therapeutics, Scientific Advisory board (4) Avexis Scientific Advisory board (5) Roche Scientific Advisory board (6) Biogen Scientific Advisory board (7) Dyne Therapeutics Scientific Advisory Board

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. (1) Neuromuscular Disorders, Editorial Board; (2) Neuropaediatrics, Editorial Board

    Patents:

    1. NONE

    Publishing Royalties:

    1. Duchenne muscular dystrophy. Oxford University Press, 2003

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. Pfizer Rare Disease SAB 2014-current Dyne Therapeutics, 2020- current

    Speakers' Bureaus:

    1. Sarepta; PTC; Roche; Biogen; Avexis

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. European Commission for the collaborative grant with Sarepta on developing Golodirsen Investigator initiated study at UCL funded by Sarepta on muscle biopsies in Golodirsen treated patients. Biogen for SMA REACH UK registry

    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

  16. Mary M. Reilly, MD, FRCP,
    17. Scientific Advisory Boards:

    1. Commercial 1. Inflectis scientific advisory board 2. Eidos steering committee

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. (1) Neuromuscular Disorders, Editorial Board (current) (2) European Journal of neurology, editorial board (current) (3) editorial board JNNP (current)

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. (1)Commercial entity: Occasional consultancy for Alynam, Inflectis, IONIS and Akcea

    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 grantS U54NS065712, 1UOINS109403-01, R21TR003034 MRC grant MR/S005021/1

    Research Support, Academic Entities:

    1. • MDA grant (MDA510281) Muscle MRI in Charcot Marie Tooth disease (PI) 2017-2021 CMTA grants for CMT1B,2A and 1X

    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

  17. Richard S. Finkel, MD,
    18. Scientific Advisory Boards:

    1. (1) SMA REACH, external advisor (2) SMA Europe, scientific advisory board (3) AveXis advisory board and DSMB member (4) Biogen advisory board (5) Roche advisory board (6) Catabasis advisory board (7) Scholar Rock advisory board (8) Sarepta advisory board (9) Neurogene advisory board (10) n-Lorem Foundation advisory board (11) Every Life Foundation advisory board

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. (1) Cure SMA: funding for travel to speak at conferences, 2005-19 (2) SMA Foundation: funding for travel to attend workshops, 2007-17 (3) Muscular Dystrophy Assn: funding for travel to conferences, 2011-19 (4) Biogen: speaker honoraria, funding for travel to speak at conferences and with regulatory authorities, 2013-19 (5) AveXis: speaker honoraria, funding for travel to speak at conferences and with regulatory authorities, 2015-17 (6) Roche: speaker honoraria, funding for travel to speak at conferences and with regulatory authorities, 2013-19 (7) Catabasis: funding for travel to speak with regulatory authorities, 2015-17

    Editorial Boards:

    1. Associate Editor of textbook of Principles and Practice of Pediatric Neurology Editorial advisory board member (1) Neuromuscular Disorders, 2010- (2) J. Neuromuscular Diseases, 2013- Spouse serves on the editorial board of Arthritis Research and Therapy without compensation, 1999-

    Patents:

    1. Patents held by spouse: 1. Finkel, T.H., Kubo, R.: Mouse Anti-TCR Zeta Antibody to Southern Biotechnology Associates. 2. Finkel, T.H., Kubo, R.: Anti-Human TCR Zeta mAb to Upstate Pharmaceuticals, NY. 3. Finkel, T.H., Kubo, R.: Anti- Human TCR Zeta mAb to Santa Cruz Biotechnology.

    Publishing Royalties:

    1. Swaiman's Principles and Practice of Pediatric Neurology, 6th edition; Elsevier, 2017, royalties received 2018-20

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. (1) Capricor: 2017-19 (2) Biogen: 2014-21 (3) Roche: 2015-21 (4) AveXis: 2015-20 (5) Novartis: 2015-21

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical procedures or imaging studies:

    1. Approximately 10% of my time and effort is devoted to patient care at Le Bonheur Children's Hospital, mainly children with neuromuscular disorders. Approximately 70% effort is involved with clinical research and trials. The remainder of my time is spent in teaching and administrative activities.

    Research Support, Commercial Entities:

    1. Dr. Richard Finkel: (1) Capricor: advisor, investigator, 2017-19 (2) Biogen: advisor, investigator 2014-21 (3) ReveraGen: advisor, investigator, 2016-21 (4) Cytokinetics: investigator, 2016-18 (5) AveXis: advisor, investigator, 2016-20 (7) Roche: advisor, investigator, 2016-21 (8) Sarepta: investigator, 2015-18 (9) Catabasis: advisor, investigator, 2017-21

    Research Support, Government Entities:

    1. (1) 5U01NS061799, Co-I, 2010-19 (2) RO1AR056973, Co-I, 2010-19 (3) 1U54NS065712-01, Co-I, 2009-19

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. (1) Spinal Muscular Atrophy Foundation (2) Cure SMA (3) Muscular Dystrophy Association (5) Charcot-Marie-Tooth Association (6) n-Lorem Foundation (7) Every Life Foundation

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License fee payments, Technology or Inventions:

    1. Licensing fees from Children's Hospital of Philadelphia for the CHOP INTEND, 2018-21 Spouse receives licensing fee payments: 1. Finkel, T.H., Kubo, R.: Mouse Anti-TCR Zeta Antibody to Southern Biotechnology Associates. 2. Finkel, T.H., Kubo, R.: Anti-Human TCR Zeta mAb to Upstate Pharmaceuticals, NY. 3. Finkel, T.H., Kubo, R.: Anti-Human TCR Zeta mAb to Santa Cruz Biotechnology.

    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

  18. Janet E. Sowden, BSc,
    19. 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. (1)The Inherited Neuropathies Consortium Rare Disease Clinical Research Network, National Institute of Neurological Disorders and Stroke (U54NS065712) Study Coordinator, 2010-present (2)NINDS, 1U01NS109403-01, Project Manager, 2019-present

    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

  19. Katy J. Eichinger, PhD,
    20. Scientific Advisory Boards:

    1. (1) Advisory board for Biogen, Acceleron, PTC Therapeutics

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Funding for travel from the (1) FSH Society and the (2) Myotonic Dystrophy Foundation

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. Consultant fees from (1) Ionis Pharmaceuticals,(2) Biogen, (3)Acceleron Pharmaceuticals (4) Fulcrum Therapeutics (5) Avidity

    Speakers' Bureaus:

    1. NONE

    Other activities:

    1. Ology- webcast panelist

    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. Charcot Marie Tooth Association

    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

  20. David N. Herrmann, MBBCh,
    21. Scientific Advisory Boards:

    1. unrelated to the present study (1)Acceleron, Inc - unrelated to present study. (2)Neurogene (Unrelated) (3)Regenacy (Unrelated)

    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. Preceding 24 months. All unrelated. (1) Acceleron Pharma(Consultant. Unrelated to present manuscript) (2) Guidepoint Global (3) Gerson Lehrman Group (4) Slingshot Insights (5) Clear View Health Partners (6) Trinity Partners (7) Cydan (8) Schlesinger (11) Sarepta (Unrelated) (12)Neurogene (unrelated to present manuscript)

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical procedures or imaging studies:

    1. Although this is not a study guideline or practice parameter I disclose that I direct an academic, clinical skin biopsy laboratory for assessment of epidermal nerve fiber density and small fiber neuropathy diagnosis. Approximately 20% effort.

    Research Support, Commercial Entities:

    1. Site PI. A Phase 2 Randomized, Double-Blind, Placebo- Controlled Study of ACE-083 in Patients with Charcot- Marie Tooth Disease Types 1 and X. Acceleron Pharma, Inc.

    Research Support, Government Entities:

    1. Site PI, Inherited Neuropathy Consortium NINDS-2U54 NS065712. PI. The ACT-CMT study. NINDS. 1U01NS109403-02 Site PI. NIDDK. The Effect of high intensity interval training and surgical weight loss on distal symmetric polyneuropathy outcomes R01DK115687 Site Investigator.NeuroNEXT: Topiramate as a Disease Altering Therapy for Cryptogenic Sensory Peripheral Neuropathy (CSPN) NINDS. U01NS077265

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. Friedreich's Ataxia Research Alliance Charcot Marie Tooth Association

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License fee payments, Technology or Inventions:

    1. (1) Outcome Measure, through Copyright to University of Rochester. Unrelated to the present publication. (2) Peripheral neuropathy(mechanisms) through intellectual property of University of Rochester. Unrelated to present publication.

    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

  21. Michael E. Shy, MD,
    22. Scientific Advisory Boards:

    1. (1) Charcot Marie Tooth Association-Non Profit (2) Muscular Dystrophy Association (MDA)-Non Profit

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. Neuromuscular Disorders, Editorial Board Muscle and Nerve, Editorial Board

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. (1) Alnylam Pharmaceuticals - Consultant (2) Mitochondria in Motion-Consultant (3) Inflectis BioSci - Consultant (4) Neurogene - Consultant (5) Applied Therapeutics - (Consultant)

    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. NINDS/Office of Rare Disease/NCATS

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. (1) Muscular Dystrophy Association; (2) Charcot Marie Tooth Association

    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

  22. Joshua Burns, PhD* and
    23. 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. Work in the Burns Group is supported by the Australian Government Department of Health (NHMRC-MRFF #1152226); US National Institutes of Health (NINDS #1U01NS109403-01, NINDS #U54NS065712); Muscular Dystrophy Association (Idea Grant #876246); Charcot-Marie Tooth Association of Australia; Charcot Marie Tooth Association (USA); Cerebral Palsy Alliance; Diabetes Australia; Elizabeth Lottie May Rosenthal Bone Bequest; Humpty Dumpty Foundation; Kids Neuroscience Centre, The University of Sydney.

    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

  23. Manoj P. Menezes, MD, PhD*
    24. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. (1) Journal of Paediatrics and Child Health, journal editor, 2017-2021 (2) Journal of the Peripheral Nervous System, member of editorial board, 2017-2021

    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. CureRTD 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

  1. From the University of Sydney (G.A.D., S.P.G., M.P.M.), Faculty of Medicine and Health; Children's Hospital at Westmead (G.A.D., S.P.G., K.M.D.C., J.B., M.P.M.); University of Sydney (K.M.D.C., M.J.M., J.B.), School of Health Sciences; Faculty of Health and Medicine (J.N.B.), University of Newcastle, Australia; Departments of Pediatrics (R.R.S.) and Neurology (M.E.S.), Carver College of Medicine, University of Iowa, Iowa City; Division of Neurology (S.W.Y.) and Department of Occupational Therapy (T.E.), Children's Hospital of Philadelphia; Department of Neurology (S.W.Y., T.E.), Perelman School of Medicine, University of Pennsylvania, Philadelphia; Fondazione IRCCS Istituto Neurologico Carlo Besta (I.M., M.F., E.P., D.P.), Milan, Italy; Centre for Neuromuscular Diseases (M.L., M.M.R.), University College London, Queen Square; University College London Institute of Child Health & Great Ormond Street Hospital (T.B., F.M.), London, England; Translational Neurosciences (Pediatrics) (R.S.F.), St. Jude Children's Research Hospital, Memphis, TN; and Department of Neurology (J.E.S., K.J.E., D.N.H.), University of Rochester, NY.
  1. Correspondence
    Ms. Donlevy gabrielle.donlevy{at}health.nsw.gov.au
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