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October 2021; 11 (5) Research

Long-term Safety and Efficacy of Mexiletine in Myotonic Dystrophy Types 1 and 2

Christina Mousele, Emma Matthews, Robert D.S. Pitceathly, Michael G. Hanna, Susan MacDonald, Konstantinos Savvatis, Aisling Carr, Christopher Turner
First published March 24, 2021, DOI: https://doi.org/10.1212/CPJ.0000000000001073
Christina Mousele
UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery (CM); Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery (EM, RDSP, MGH, AC, CT); The National Hospital for Neurology and Neurosurgery (SM), Atkinson-Morley Neuromuscular Centre (EM); and Inherited Cardiovascular Diseases Unit, Barts Health Centre, Barts Healthcare NHS Trust (KS), London, United Kingdom.
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Emma Matthews
UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery (CM); Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery (EM, RDSP, MGH, AC, CT); The National Hospital for Neurology and Neurosurgery (SM), Atkinson-Morley Neuromuscular Centre (EM); and Inherited Cardiovascular Diseases Unit, Barts Health Centre, Barts Healthcare NHS Trust (KS), London, United Kingdom.
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Robert D.S. Pitceathly
UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery (CM); Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery (EM, RDSP, MGH, AC, CT); The National Hospital for Neurology and Neurosurgery (SM), Atkinson-Morley Neuromuscular Centre (EM); and Inherited Cardiovascular Diseases Unit, Barts Health Centre, Barts Healthcare NHS Trust (KS), London, United Kingdom.
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Michael G. Hanna
UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery (CM); Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery (EM, RDSP, MGH, AC, CT); The National Hospital for Neurology and Neurosurgery (SM), Atkinson-Morley Neuromuscular Centre (EM); and Inherited Cardiovascular Diseases Unit, Barts Health Centre, Barts Healthcare NHS Trust (KS), London, United Kingdom.
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Susan MacDonald
UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery (CM); Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery (EM, RDSP, MGH, AC, CT); The National Hospital for Neurology and Neurosurgery (SM), Atkinson-Morley Neuromuscular Centre (EM); and Inherited Cardiovascular Diseases Unit, Barts Health Centre, Barts Healthcare NHS Trust (KS), London, United Kingdom.
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Konstantinos Savvatis
UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery (CM); Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery (EM, RDSP, MGH, AC, CT); The National Hospital for Neurology and Neurosurgery (SM), Atkinson-Morley Neuromuscular Centre (EM); and Inherited Cardiovascular Diseases Unit, Barts Health Centre, Barts Healthcare NHS Trust (KS), London, United Kingdom.
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Aisling Carr
UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery (CM); Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery (EM, RDSP, MGH, AC, CT); The National Hospital for Neurology and Neurosurgery (SM), Atkinson-Morley Neuromuscular Centre (EM); and Inherited Cardiovascular Diseases Unit, Barts Health Centre, Barts Healthcare NHS Trust (KS), London, United Kingdom.
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Christopher Turner
UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery (CM); Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery (EM, RDSP, MGH, AC, CT); The National Hospital for Neurology and Neurosurgery (SM), Atkinson-Morley Neuromuscular Centre (EM); and Inherited Cardiovascular Diseases Unit, Barts Health Centre, Barts Healthcare NHS Trust (KS), London, United Kingdom.
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Full PDF
Citation
Long-term Safety and Efficacy of Mexiletine in Myotonic Dystrophy Types 1 and 2
Christina Mousele, Emma Matthews, Robert D.S. Pitceathly, Michael G. Hanna, Susan MacDonald, Konstantinos Savvatis, Aisling Carr, Christopher Turner
Neurol Clin Pract Oct 2021, 11 (5) e682-e685; DOI: 10.1212/CPJ.0000000000001073

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Article Information

vol. 11 no. 5 e682-e685
DOI: 
https://doi.org/10.1212/CPJ.0000000000001073

Published By: 
Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology
Print ISSN: 
2163-0402
Online ISSN: 
2163-0933
History: 
  • Received July 29, 2020
  • Accepted February 10, 2021
  • First Published March 24, 2021.

Article Versions

  • Previous version (March 24, 2021 - 12:45).
  • You are viewing the most recent version of this article.
Copyright & Usage: 
© 2021 American Academy of Neurology

Author Disclosures

    1. Christina Mousele, MD, MSc,
    2. Emma Matthews, PhD,
    3. Robert D.S. Pitceathly, MD, PhD,
    4. Michael G. Hanna, FMedSci,
    5. Susan MacDonald, BN,
    6. Konstantinos Savvatis, PhD,
    7. Aisling Carr, PhD and
    8. Christopher Turner, PhD
  1. Christina Mousele, MD, MSc,
  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. 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. Emma Matthews, PhD,
  4. Scientific Advisory Boards:
    1. NONE

    Gifts:
    1. NONE

    Funding for Travel or Speaker Honoraria:
    1. NONE

    Editorial Boards:
    1. Neuromuscular Disorders, Member of the Editorial Board, Jan 2019 - current

    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. I was supported by a Wellcome Clinical Research Career Development Fellowship (209583) 2018-2020.

    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. Robert D.S. Pitceathly, MD, 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. 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. Michael G. Hanna, FMedSci,
  8. 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

  9. Susan MacDonald, BN,
  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

  11. Konstantinos Savvatis, PhD,
  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

  13. Aisling Carr, PhD and
  14. Scientific Advisory Boards:
    1. ADvisory role to CSL (PATH study) Advisory role to Lupin (mexiletine for myotonia)

    Gifts:
    1. NONE

    Funding for travel or speaker honoraria:
    1. Honoraria for speaking at CSL, grifols and akcea symposia

    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. Travel support from Grifols and CSL to attend conferences

    Clinical Procedures or Imaging Studies:
    1. NONE

    Research Support, Commercial Entities:
    1. NONE

    Research Support, Government Entities:
    1. UCLH BRC funding (1 PA per week)

    Research Support, Academic Entities:
    1. ULCH BRC (Biomedical research council)

    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. Christopher Turner, PhD
  16. Scientific Advisory Boards:
    1. Non-profit. Steering committee of the UK Myotonic Dystrophy National registry

    Gifts:
    1. NONE

    Funding for travel or speaker honoraria:
    1. 1) Commercial: Speaker for Genzyme

    Editorial Boards:
    1. Neuromuscular Disorders-Editorial Board

    Patents:
    1. NONE

    Publishing Royalties:
    1. NONE

    Employment, Commercial Entity:
    1. NONE

    Consultancies:
    1. Dyne Therapeutics-Commercial

    Speakers' Bureaus:
    1. NONE

    Other Activities:
    1. NONE

    Clinical Procedures or Imaging Studies:
    1. NONE

    Research Support, Commercial Entities:
    1. 1) Commercial entity: Genzyme funding for Research Nurse in Myotonic Dystrophy PHENODM1 study

    Research Support, Government Entities:
    1. NIHR support for PHENODM1 study

    Research Support, Academic Entities:
    1. Academic Entity: LCRN funding of research physiotherapist

    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. Commercial entity: Act as an expert witness for mostly cases of head injury.

  1. UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery (CM); Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery (EM, RDSP, MGH, AC, CT); The National Hospital for Neurology and Neurosurgery (SM), Atkinson-Morley Neuromuscular Centre (EM); and Inherited Cardiovascular Diseases Unit, Barts Health Centre, Barts Healthcare NHS Trust (KS), London, United Kingdom.
  1. Correspondence
    Dr. Mousele christinamousele92{at}gmail.com
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