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August 2021; 11 (4) Research

Achieving Life Milestones in Duchenne/Becker Muscular Dystrophy

A Retrospective Analysis

Andrew Donaldson, Debra Guntrum, Emma Ciafaloni, Jeffrey Statland
First published September 23, 2020, DOI: https://doi.org/10.1212/CPJ.0000000000000970
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Article Information

vol. 11 no. 4 311-317
DOI: 
https://doi.org/10.1212/CPJ.0000000000000970
Published By: 
Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology
Print ISSN: 
2163-0402
Online ISSN: 
2163-0933
History: 
  • Received December 30, 2019
  • Accepted July 30, 2020
  • First Published September 23, 2020.

Article Versions

Copyright & Usage: 
© 2021 American Academy of Neurology

Author Disclosures

    1. Andrew Donaldson,
    2. Debra Guntrum, NP,
    3. Emma Ciafaloni, MD and
    4. Jeffrey Statland, MD
  1. Andrew Donaldson,
    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

  2. Debra Guntrum, NP,
    3. Scientific Advisory Boards:

    1. (1)AveXis (2)Biogen (3)Sarepta (4)PTC

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. (1) Biogen honoraria for nursing advisory board (2) PTC honoraria for nursing advisory board

    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 Ciafaloni, MD and
    4. Scientific Advisory Boards:

    1. Sarepta Avexis Biogen Santhera PTC Wave Sanofi

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. MedLink

    Patents:

    1. NONE

    Publishing Royalties:

    1. Oxford Press

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. Center Disease Control Prevention: "Surveillance and Epidemiological Research of Duchenne and Becker Muscular Dystrophy"

    Speakers' Bureaus:

    1. Biogen

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. Sarepta, PTC, Santhera, Orphazyme: clinical trial support

    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. Jeffrey Statland, MD
    5. Scientific Advisory Boards:

    1. 1) Advisory Board - Fulcrum 2) Advisory Board - Sarepta 3) Advisory Board - Acceleron 4) Advisory Board - Dyne

    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) Acceleron 2) Fulcrum 3) Strongbridge 4) Dyne 5) Sarepta 6) Avidity

    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) NINDS 5U01NS101944-02, Role co-PI, 6/2017-present 2) NCATS R21TR003184-01, Role co-PI, 6/2020-present

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. 1) FSH Society Grant - FSHD clinical trial research network 2) MDA Infrastructure Grant - 06/2018 - present 3) Friends of FSH Research - MOVE FSHD study

    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. University of Kansas Medical Center (AD, JS); and University of Rochester Medical Center (DG, EC), NY.
  1. Correspondence
    Dr. Statland JStatland{at}kumc.edu
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