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June 2021; 11 (3) ResearchOpen Access

Nusinersen Treatment in Adults With Spinal Muscular Atrophy

Tina Duong, Connie Wolford, Michael P. McDermott, Chelsea E. Macpherson, Amy Pasternak, Allan M. Glanzman, William B. Martens, Elizabeth Kichula, Basil T. Darras, Darryl C. De Vivo, View ORCID ProfileZarazuela Zolkipli-Cunningham, View ORCID ProfileRichard S. Finkel, View ORCID ProfileMichael Zeineh, Max Wintermark, Jacinda Sampson, Katharine A. Hagerman, Sally Dunaway Young, John W. Day
First published January 25, 2021, DOI: https://doi.org/10.1212/CPJ.0000000000001033
Tina Duong
Departments of Neurology (TD, CW, SDY, KAH, JS, JWD), Rehabilitation (CEM), and Radiology (MZ, MW), Stanford University School of Medicine, Palo Alto, CA; Departments of Biostatistics and Computational Biology (MPM), and Neurology (MPM and WBM), University of Rochester Medical Center, NY; Department of Neurology (AP,BTD), Boston Children's Hospital, MA; Department of Neurology (DCD), Columbia University, New York; Department of Neurology (AMG EAK, ZZ) Children's Hospital of Philadelphia, PA; and Department of Pediatrics (RSF) Nemours Children's Hospital, Orlando, FL.
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Connie Wolford
Departments of Neurology (TD, CW, SDY, KAH, JS, JWD), Rehabilitation (CEM), and Radiology (MZ, MW), Stanford University School of Medicine, Palo Alto, CA; Departments of Biostatistics and Computational Biology (MPM), and Neurology (MPM and WBM), University of Rochester Medical Center, NY; Department of Neurology (AP,BTD), Boston Children's Hospital, MA; Department of Neurology (DCD), Columbia University, New York; Department of Neurology (AMG EAK, ZZ) Children's Hospital of Philadelphia, PA; and Department of Pediatrics (RSF) Nemours Children's Hospital, Orlando, FL.
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  • For correspondence: cwolford@stanfordhealthcare.org
Michael P. McDermott
Departments of Neurology (TD, CW, SDY, KAH, JS, JWD), Rehabilitation (CEM), and Radiology (MZ, MW), Stanford University School of Medicine, Palo Alto, CA; Departments of Biostatistics and Computational Biology (MPM), and Neurology (MPM and WBM), University of Rochester Medical Center, NY; Department of Neurology (AP,BTD), Boston Children's Hospital, MA; Department of Neurology (DCD), Columbia University, New York; Department of Neurology (AMG EAK, ZZ) Children's Hospital of Philadelphia, PA; and Department of Pediatrics (RSF) Nemours Children's Hospital, Orlando, FL.
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  • For correspondence: michael_mcdermott@urmc.rochester.edu
Chelsea E. Macpherson
Departments of Neurology (TD, CW, SDY, KAH, JS, JWD), Rehabilitation (CEM), and Radiology (MZ, MW), Stanford University School of Medicine, Palo Alto, CA; Departments of Biostatistics and Computational Biology (MPM), and Neurology (MPM and WBM), University of Rochester Medical Center, NY; Department of Neurology (AP,BTD), Boston Children's Hospital, MA; Department of Neurology (DCD), Columbia University, New York; Department of Neurology (AMG EAK, ZZ) Children's Hospital of Philadelphia, PA; and Department of Pediatrics (RSF) Nemours Children's Hospital, Orlando, FL.
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  • For correspondence: cem2183@gmail.com
Amy Pasternak
Departments of Neurology (TD, CW, SDY, KAH, JS, JWD), Rehabilitation (CEM), and Radiology (MZ, MW), Stanford University School of Medicine, Palo Alto, CA; Departments of Biostatistics and Computational Biology (MPM), and Neurology (MPM and WBM), University of Rochester Medical Center, NY; Department of Neurology (AP,BTD), Boston Children's Hospital, MA; Department of Neurology (DCD), Columbia University, New York; Department of Neurology (AMG EAK, ZZ) Children's Hospital of Philadelphia, PA; and Department of Pediatrics (RSF) Nemours Children's Hospital, Orlando, FL.
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  • For correspondence: amy.pasternak@childrens.harvard.edu
Allan M. Glanzman
Departments of Neurology (TD, CW, SDY, KAH, JS, JWD), Rehabilitation (CEM), and Radiology (MZ, MW), Stanford University School of Medicine, Palo Alto, CA; Departments of Biostatistics and Computational Biology (MPM), and Neurology (MPM and WBM), University of Rochester Medical Center, NY; Department of Neurology (AP,BTD), Boston Children's Hospital, MA; Department of Neurology (DCD), Columbia University, New York; Department of Neurology (AMG EAK, ZZ) Children's Hospital of Philadelphia, PA; and Department of Pediatrics (RSF) Nemours Children's Hospital, Orlando, FL.
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  • For correspondence: glanzmana@email.chop.edu
William B. Martens
Departments of Neurology (TD, CW, SDY, KAH, JS, JWD), Rehabilitation (CEM), and Radiology (MZ, MW), Stanford University School of Medicine, Palo Alto, CA; Departments of Biostatistics and Computational Biology (MPM), and Neurology (MPM and WBM), University of Rochester Medical Center, NY; Department of Neurology (AP,BTD), Boston Children's Hospital, MA; Department of Neurology (DCD), Columbia University, New York; Department of Neurology (AMG EAK, ZZ) Children's Hospital of Philadelphia, PA; and Department of Pediatrics (RSF) Nemours Children's Hospital, Orlando, FL.
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  • For correspondence: bill_martens@urmc.rochester.edu
Elizabeth Kichula
Departments of Neurology (TD, CW, SDY, KAH, JS, JWD), Rehabilitation (CEM), and Radiology (MZ, MW), Stanford University School of Medicine, Palo Alto, CA; Departments of Biostatistics and Computational Biology (MPM), and Neurology (MPM and WBM), University of Rochester Medical Center, NY; Department of Neurology (AP,BTD), Boston Children's Hospital, MA; Department of Neurology (DCD), Columbia University, New York; Department of Neurology (AMG EAK, ZZ) Children's Hospital of Philadelphia, PA; and Department of Pediatrics (RSF) Nemours Children's Hospital, Orlando, FL.
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  • For correspondence: kichulae@email.chop.edu
Basil T. Darras
Departments of Neurology (TD, CW, SDY, KAH, JS, JWD), Rehabilitation (CEM), and Radiology (MZ, MW), Stanford University School of Medicine, Palo Alto, CA; Departments of Biostatistics and Computational Biology (MPM), and Neurology (MPM and WBM), University of Rochester Medical Center, NY; Department of Neurology (AP,BTD), Boston Children's Hospital, MA; Department of Neurology (DCD), Columbia University, New York; Department of Neurology (AMG EAK, ZZ) Children's Hospital of Philadelphia, PA; and Department of Pediatrics (RSF) Nemours Children's Hospital, Orlando, FL.
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  • For correspondence: basil.darras@childrens.harvard.edu
Darryl C. De Vivo
Departments of Neurology (TD, CW, SDY, KAH, JS, JWD), Rehabilitation (CEM), and Radiology (MZ, MW), Stanford University School of Medicine, Palo Alto, CA; Departments of Biostatistics and Computational Biology (MPM), and Neurology (MPM and WBM), University of Rochester Medical Center, NY; Department of Neurology (AP,BTD), Boston Children's Hospital, MA; Department of Neurology (DCD), Columbia University, New York; Department of Neurology (AMG EAK, ZZ) Children's Hospital of Philadelphia, PA; and Department of Pediatrics (RSF) Nemours Children's Hospital, Orlando, FL.
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  • For correspondence: dcd1@cumc.columbia.edu
Zarazuela Zolkipli-Cunningham
Departments of Neurology (TD, CW, SDY, KAH, JS, JWD), Rehabilitation (CEM), and Radiology (MZ, MW), Stanford University School of Medicine, Palo Alto, CA; Departments of Biostatistics and Computational Biology (MPM), and Neurology (MPM and WBM), University of Rochester Medical Center, NY; Department of Neurology (AP,BTD), Boston Children's Hospital, MA; Department of Neurology (DCD), Columbia University, New York; Department of Neurology (AMG EAK, ZZ) Children's Hospital of Philadelphia, PA; and Department of Pediatrics (RSF) Nemours Children's Hospital, Orlando, FL.
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  • ORCID record for Zarazuela Zolkipli-Cunningham
  • For correspondence: zolkipliz@email.chop.edu
Richard S. Finkel
Departments of Neurology (TD, CW, SDY, KAH, JS, JWD), Rehabilitation (CEM), and Radiology (MZ, MW), Stanford University School of Medicine, Palo Alto, CA; Departments of Biostatistics and Computational Biology (MPM), and Neurology (MPM and WBM), University of Rochester Medical Center, NY; Department of Neurology (AP,BTD), Boston Children's Hospital, MA; Department of Neurology (DCD), Columbia University, New York; Department of Neurology (AMG EAK, ZZ) Children's Hospital of Philadelphia, PA; and Department of Pediatrics (RSF) Nemours Children's Hospital, Orlando, FL.
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  • ORCID record for Richard S. Finkel
  • For correspondence: richard.finkel@stjude.org
Michael Zeineh
Departments of Neurology (TD, CW, SDY, KAH, JS, JWD), Rehabilitation (CEM), and Radiology (MZ, MW), Stanford University School of Medicine, Palo Alto, CA; Departments of Biostatistics and Computational Biology (MPM), and Neurology (MPM and WBM), University of Rochester Medical Center, NY; Department of Neurology (AP,BTD), Boston Children's Hospital, MA; Department of Neurology (DCD), Columbia University, New York; Department of Neurology (AMG EAK, ZZ) Children's Hospital of Philadelphia, PA; and Department of Pediatrics (RSF) Nemours Children's Hospital, Orlando, FL.
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  • ORCID record for Michael Zeineh
Max Wintermark
Departments of Neurology (TD, CW, SDY, KAH, JS, JWD), Rehabilitation (CEM), and Radiology (MZ, MW), Stanford University School of Medicine, Palo Alto, CA; Departments of Biostatistics and Computational Biology (MPM), and Neurology (MPM and WBM), University of Rochester Medical Center, NY; Department of Neurology (AP,BTD), Boston Children's Hospital, MA; Department of Neurology (DCD), Columbia University, New York; Department of Neurology (AMG EAK, ZZ) Children's Hospital of Philadelphia, PA; and Department of Pediatrics (RSF) Nemours Children's Hospital, Orlando, FL.
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  • For correspondence: mzeineh@stanford.edu
Jacinda Sampson
Departments of Neurology (TD, CW, SDY, KAH, JS, JWD), Rehabilitation (CEM), and Radiology (MZ, MW), Stanford University School of Medicine, Palo Alto, CA; Departments of Biostatistics and Computational Biology (MPM), and Neurology (MPM and WBM), University of Rochester Medical Center, NY; Department of Neurology (AP,BTD), Boston Children's Hospital, MA; Department of Neurology (DCD), Columbia University, New York; Department of Neurology (AMG EAK, ZZ) Children's Hospital of Philadelphia, PA; and Department of Pediatrics (RSF) Nemours Children's Hospital, Orlando, FL.
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  • For correspondence: jacindas@stanford.edu
Katharine A. Hagerman
Departments of Neurology (TD, CW, SDY, KAH, JS, JWD), Rehabilitation (CEM), and Radiology (MZ, MW), Stanford University School of Medicine, Palo Alto, CA; Departments of Biostatistics and Computational Biology (MPM), and Neurology (MPM and WBM), University of Rochester Medical Center, NY; Department of Neurology (AP,BTD), Boston Children's Hospital, MA; Department of Neurology (DCD), Columbia University, New York; Department of Neurology (AMG EAK, ZZ) Children's Hospital of Philadelphia, PA; and Department of Pediatrics (RSF) Nemours Children's Hospital, Orlando, FL.
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  • For correspondence: khagerma@stanford.edu
Sally Dunaway Young
Departments of Neurology (TD, CW, SDY, KAH, JS, JWD), Rehabilitation (CEM), and Radiology (MZ, MW), Stanford University School of Medicine, Palo Alto, CA; Departments of Biostatistics and Computational Biology (MPM), and Neurology (MPM and WBM), University of Rochester Medical Center, NY; Department of Neurology (AP,BTD), Boston Children's Hospital, MA; Department of Neurology (DCD), Columbia University, New York; Department of Neurology (AMG EAK, ZZ) Children's Hospital of Philadelphia, PA; and Department of Pediatrics (RSF) Nemours Children's Hospital, Orlando, FL.
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  • For correspondence: sdy@stanford.edu
John W. Day
Departments of Neurology (TD, CW, SDY, KAH, JS, JWD), Rehabilitation (CEM), and Radiology (MZ, MW), Stanford University School of Medicine, Palo Alto, CA; Departments of Biostatistics and Computational Biology (MPM), and Neurology (MPM and WBM), University of Rochester Medical Center, NY; Department of Neurology (AP,BTD), Boston Children's Hospital, MA; Department of Neurology (DCD), Columbia University, New York; Department of Neurology (AMG EAK, ZZ) Children's Hospital of Philadelphia, PA; and Department of Pediatrics (RSF) Nemours Children's Hospital, Orlando, FL.
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  • For correspondence: jwday@stanford.edu
Full PDF
Citation
Nusinersen Treatment in Adults With Spinal Muscular Atrophy
Tina Duong, Connie Wolford, Michael P. McDermott, Chelsea E. Macpherson, Amy Pasternak, Allan M. Glanzman, William B. Martens, Elizabeth Kichula, Basil T. Darras, Darryl C. De Vivo, Zarazuela Zolkipli-Cunningham, Richard S. Finkel, Michael Zeineh, Max Wintermark, Jacinda Sampson, Katharine A. Hagerman, Sally Dunaway Young, John W. Day
Neurol Clin Pract Jun 2021, 11 (3) e317-e327; DOI: 10.1212/CPJ.0000000000001033

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Article Information

vol. 11 no. 3 e317-e327
DOI: 
https://doi.org/10.1212/CPJ.0000000000001033

Published By: 
Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology
Print ISSN: 
2163-0402
Online ISSN: 
2163-0933
History: 
  • Received April 9, 2020
  • Accepted November 3, 2020
  • First Published January 25, 2021.

Article Versions

  • Previous version (January 25, 2021 - 12:45).
  • You are viewing the most recent version of this article.
Copyright & Usage: 
Copyright © 2021 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology. This is an open access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives License 4.0 (CC BY-NC-ND), which permits downloading and sharing the work provided it is properly cited. The work cannot be changed in any way or used commercially without permission from the journal.

Author Disclosures

    1. Tina Duong, PT, PhD,
    2. Connie Wolford, MSN, FNP-BC (cwolford{at}stanfordhealthcare.org),
    3. Michael P. McDermott, PhD (michael_mcdermott{at}urmc.rochester.edu),
    4. Chelsea E. Macpherson, PT, DPT, NCS (cem2183{at}gmail.com),
    5. Amy Pasternak, PT, DPT, PCS (amy.pasternak{at}childrens.harvard.edu),
    6. Allan M. Glanzman, PT, DPT, PCS (glanzmana{at}email.chop.edu),
    7. William B. Martens, BA (bill_martens{at}urmc.rochester.edu),
    8. Elizabeth Kichula, MD, BA (kichulae{at}email.chop.edu),
    9. Basil T. Darras, MD (basil.darras{at}childrens.harvard.edu),
    10. Darryl C. De Vivo, MD (dcd1{at}cumc.columbia.edu),
    11. Zarazuela Zolkipli-Cunningham, MBChB, MRCP (zolkipliz{at}email.chop.edu),
    12. Richard S. Finkel, MD (richard.finkel{at}stjude.org),
    13. Michael Zeineh, MD, PhD,
    14. Max Wintermark, MD (mzeineh{at}stanford.edu),
    15. Jacinda Sampson, MD, PhD (jacindas{at}stanford.edu),
    16. Katharine A. Hagerman, PhD (khagerma{at}stanford.edu),
    17. Sally Dunaway Young, PT, DPT (sdy{at}stanford.edu) and
    18. John W. Day, MD, PhD on behalf of the Stanford Neuromuscular Coordinating Center (jwday{at}stanford.edu)
  1. Tina Duong, PT, PhD,
  2. Scientific Advisory Boards:
    1. Advisory board or consultancy for 1) Commercial: Roche, Novartis, Biogen, Scholar Rock 2) Non-Profit: SMA foundation

    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) Commercial: Biogen, Roche, Genentech, Trinds, ATOM International

    Speakers' Bureaus:
    1. 1)Commercial: Genentech, Roche, Biogen 2) CureSMA

    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) SMA Foundation

    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. Connie Wolford, MSN, FNP-BC (cwolford{at}stanfordhealthcare.org),
  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. Biogen speaker's bureau

    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. Michael P. McDermott, PhD (michael_mcdermott{at}urmc.rochester.edu),
  6. Scientific Advisory Boards:
    1. DSMB service for AstraZeneca, Eli Lilly and Company, aTyr Pharma, NIH; Massachusetts General Hospital; Catabasis Pharmaceuticals, Inc.; Vaccinex, Inc.; Cynapsus Therapeutics; Voyager Therapeutics; and Prilenia Therapeutics Development, Ltd.

    Gifts:
    1. NONE

    Funding for Travel or Speaker Honoraria:
    1. None

    Editorial Boards:
    1. Movement Disorders, Editorial Board, 2010-2020; Chance, Editor, 2014-2019.

    Patents:
    1. NONE

    Publishing Royalties:
    1. NONE

    Employment, Commercial Entity:
    1. NONE

    Consultancies:
    1. Received consulting fees from Fulcrum Therapeutics, Inc.

    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, EY025990, Co-I, 2017-2019; NIH, NS080840, Co-I, 2014-2019; FDA, FD005936, Co-I, 2016-present; FDA, FD004809, Co-I, 2015-present; NIH, NS108676, Co-I, 2018-present; NIH, NS105479, Co-I, 2018-present; NIH, NS109403, Co-I, 2019-present; NIH, MH119264, Co-I, 2019-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

  7. Chelsea E. Macpherson, PT, DPT, NCS (cem2183{at}gmail.com),
  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. Amy Pasternak, PT, DPT, PCS (amy.pasternak{at}childrens.harvard.edu),
  10. Scientific Advisory Boards:
    1. 1. Scholar Rock 2. Biogen

    Gifts:
    1. NONE

    Funding for travel or speaker honoraria:
    1. 1. Biogen 2. Stanford CME

    Editorial Boards:
    1. NONE

    Patents:
    1. NONE

    Publishing Royalties:
    1. NONE

    Employment, Commercial Entity:
    1. NONE

    Consultancies:
    1. NONE

    Speakers' Bureaus:
    1. 1. Biogen

    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. Allan M. Glanzman, PT, DPT, PCS (glanzmana{at}email.chop.edu),
  12. Scientific Advisory Boards:
    1. NONE

    Gifts:
    1. NONE

    Funding for travel or speaker honoraria:
    1. Audentis pharmaceutical, atom international, scholar rock, biogen pharmaceutical, avexis pharmaceutical

    Editorial Boards:
    1. Editorial Board, Physical & Occupational Therapy in Pediatrics 2017-present

    Patents:
    1. NONE

    Publishing Royalties:
    1. NONE

    Employment, Commercial Entity:
    1. NONE

    Consultancies:
    1. audentes pharmaceutical, aspa pharmaceutical, biogen pharmaceutical, avexis pharmaceutical, roche pharmaceutical

    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. Adjunct lecturer Arcadia University

    Research Support, Foundations and Societies:
    1. NONE

    Stock/Stock Options/Board of Directors Compensation:
    1. NONE

    License fee payments, Technology or Inventions:
    1. CHOP INTEND royalty through employer

    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. William B. Martens, BA (bill_martens{at}urmc.rochester.edu),
  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

  15. Elizabeth Kichula, MD, BA (kichulae{at}email.chop.edu),
  16. Scientific Advisory Boards:
    1. (1) PTC Therapeutics (2) Biogen (3) Stealth BioTherapeutics (4) Avexis

    Gifts:
    1. NONE

    Funding for travel or speaker honoraria:
    1. Natera

    Editorial Boards:
    1. NONE

    Patents:
    1. NONE

    Publishing Royalties:
    1. NONE

    Employment, Commercial Entity:
    1. NONE

    Consultancies:
    1. NONE

    Speakers' Bureaus:
    1. Genentech

    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

  17. Basil T. Darras, MD (basil.darras{at}childrens.harvard.edu),
  18. Scientific Advisory Boards:
    1. (1) Hoffman LaRoche (2) Cytokinetics, Inc. (3) Vertex, Inc. (4) Sarepta Therapeutics (5) Biogen (6) AveXis (6) PTC Therapeutics (7) AveXis Ad hoc SAB member; no financial interests in these companies

    Gifts:
    1. NONE

    Funding for Travel or Speaker Honoraria:
    1. NONE

    Editorial Boards:
    1. NONE

    Patents:
    1. NONE

    Publishing Royalties:
    1. (1) Various titles, UpToDate, 1999-present (2) One videotaped presentation,

    Employment, Commercial Entity:
    1. NONE

    Consultancies:
    1. NONE

    Speakers' Bureaus:
    1. Biogen

    Other Activities:
    1. NONE

    Clinical Procedures or Imaging Studies:
    1. NONE

    Research Support, Commercial Entities:
    1. (1) PTC Therapeutics (2) Scholar Rock (3) Sarepta Therapeutics (4) Biogen (5) Summit (6) AveXis (7) Roche (8) Fibrogen (9) Santhera (10) Cytokinetics

    Research Support, Government Entities:
    1. (1) NIH/NINDS, 1U10NS077269, Site-PI/PD, 2018-2023. (2) NIH/NINDS, 5U01NS061799, FOR-DMD, Site-PI, 2012-2016. (3) NIH/NINDS via Skulpt Inc., 5RNS073188, Site-PI, 2015- 2016.

    Research Support, Academic Entities:
    1. NONE

    Research Support, Foundations and Societies:
    1. (1) SMA Foundation (2) Muscular Dystrophy Association (3) Slaney Family Fund for SMA

    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. Darryl C. De Vivo, MD (dcd1{at}cumc.columbia.edu),
  20. Scientific Advisory Boards:
    1. Biogen DEVOTE Steering Committee; AveXis RESTORE Steering Committee; ASPA Therapeutics DSMC; Roche (Genentech) Advisory Board; Scholar Rock Advisory Committee.

    Gifts:
    1. NONE

    Funding for travel or speaker honoraria:
    1. 2020 Distinquished Lecture, Mount Sinai Medical Center, New York

    Editorial Boards:
    1. NONE

    Patents:
    1. Glut1DS gene therapy

    Publishing Royalties:
    1. Neuromuscular Disorders in Infancy, Childhood and Adolescence, second edition, 2015 Academic Press

    Employment, Commercial Entity:
    1. NONE

    Consultancies:
    1. Commercial Entities: Unpaid consultant for AveXis, Biogen, Ionis, Roche, Sarepta, Scholar Rock, Ultragenyx, PTC, Santhera, Mallinkrodt. Non-profit Entities: Unpaid consultant for Hope for Children Research Foundation, Glut1 Deficiency Foundation, Canavan Foundation.

    Speakers' Bureaus:
    1. NONE

    Other Activities:
    1. NONE

    Clinical Procedures or Imaging Studies:
    1. NONE

    Research Support, Commercial Entities:
    1. Principal Investigator for Clinical Trials funded by Biogen, Sarepta, PTC, Ultragenyx, Scholar Rock, GliaPharm, Fulcrum Therapeutics and performed at Columbia University.

    Research Support, Government Entities:
    1. DoD Program Project 2020-2024 focusing on treatments for mitochondrial diseases.

    Research Support, Academic Entities:
    1. NONE

    Research Support, Foundations and Societies:
    1. Hope for Children Research Foundation; Glut1 Deficiency 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

  21. Zarazuela Zolkipli-Cunningham, MBChB, MRCP (zolkipliz{at}email.chop.edu),
  22. Scientific Advisory Boards:
    1. NONE

    Gifts:
    1. NONE

    Funding for Travel or Speaker Honoraria:
    1. NONE

    Editorial Boards:
    1. NONE

    Patents:
    1. 1. Oxygen Nanosensor to measure tissue Oxygen levels. 1065-18. 2. Methods of Treating Mitochondrial Dysfunction. 62/689.

    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. NICHD U24 Leigh syndrome gene curation grant support.

    Research Support, Foundations and Societies:
    1. Pediatric Neuromuscular Clinical Research Network support from the Spinal Muscular Atrophy Foundation (SMAF)and CureSMA.

    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. Richard S. Finkel, MD (richard.finkel{at}stjude.org),
  24. Scientific Advisory Boards:
    1. (1) SMA REACH, external advisor (2) SMA Europe, scientific advisory board (3) AveXis advisory board (4) Biogen advisory board (5) Roche advisory board (6) Catabasis advisory board (7) Scholar Rock advisory board (8) Catabasis 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 on 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: 2014-20 (3) Roche: 2015-20 (4) AveXis: 2015-20 (5) Novartis: 2015-20

    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 2014-20 (3) ReveraGen: advisor, investigator, 2016-20 (4) Cytokinetics: investigator, 2016-18 (5) AveXis: advisor, investigator, 2016-20 (7) Roche: advisor, investigator, 2016-20 (8) Sarepta: investigator, 2015-18 (9) Catabasis: advisor, investigator, 2017-20

    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, 2018-20 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

  25. Michael Zeineh, MD, PhD,
  26. 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) consultant for Biogen.

    Speakers' Bureaus:
    1. NONE

    Other Activities:
    1. NONE

    Clinical Procedures or Imaging Studies:
    1. NONE

    Research Support, Commercial Entities:
    1. (1) GE Healthcare

    Research Support, Government Entities:
    1. (1) NIH/NIA, 1R01AG061120-01, (PI Zeineh), 3/2019 – 12/2023

    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

  27. Max Wintermark, MD (mzeineh{at}stanford.edu),
  28. Scientific Advisory Boards:
    1. (1) Commercial entity: Nines; (2) Commercial entity: Icometrix; (3) Commercial entity: Magnetic Insight; (4) Commercial entity: MoreHealth; (5) Commercial entity: Subtle Medical

    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

  29. Jacinda Sampson, MD, PhD (jacindas{at}stanford.edu),
  30. Scientific Advisory Boards:
    1. 1. non-profit entity (Dr Clemens, U Pittsburgh, DSMB) 2. commercial (Dyne Therapeutics, scientific advisory board) 3. commercial (Expansion Therapeutics, scientific advisory board). 4. commerical (Viking Therapeutics, scientific advisory board)

    Gifts:
    1. NONE

    Funding for travel or speaker honoraria:
    1. Funding for travel to MDF 2019 annual meeting

    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. Expert testimony, not related to industry

    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. Marigold Foundation (Myotonic dystrophy 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

  31. Katharine A. Hagerman, PhD (khagerma{at}stanford.edu),
  32. 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. (1) Biogen Inc

    Research Support, Government Entities:
    1. NONE

    Research Support, Academic Entities:
    1. NONE

    Research Support, Foundations and Societies:
    1. (1) SMA Foundation, (2) Marigold 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

  33. Sally Dunaway Young, PT, DPT (sdy{at}stanford.edu) and
  34. Scientific Advisory Boards:
    1. (1) Biogen (2) Roche/Genentech (3) Scholar Rock

    Gifts:
    1. NONE

    Funding for travel or speaker honoraria:
    1. (1) Roche/Genentech

    Editorial Boards:
    1. NONE

    Patents:
    1. NONE

    Publishing Royalties:
    1. NONE

    Employment, Commercial Entity:
    1. NONE

    Consultancies:
    1. (1) Biogen (2) Cure SMA (3) Scholar Rock (4) Roche/Genentech

    Speakers' Bureaus:
    1. NONE

    Other Activities:
    1. NONE

    Clinical Procedures or Imaging Studies:
    1. NONE

    Research Support, Commercial Entities:
    1. (1) Biogen

    Research Support, Government Entities:
    1. NONE

    Research Support, Academic Entities:
    1. NONE

    Research Support, Foundations and Societies:
    1. (1) SMA Foundation (2) Cure SMA

    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

  35. John W. Day, MD, PhD on behalf of the Stanford Neuromuscular Coordinating Center (jwday{at}stanford.edu)
  36. Scientific Advisory Boards:
    1. 1 - FOR-DMD DSMB Chair - evaluation of steroid dosing in Duchenne muscular dystrophy, Funded by NIH, PPMD, Marathon and PTC Pharmaceuticals. 2) Biogen for scientific board regarding SMA and nusinersen 3) Roche for scientific board regarding SMA and risdiplam 4) Cytokinetics for consulting regarding SMA and CK-107 5) AveXis/Novartis for consulting regarding SMA and AVXS101/branaplam 6) Pfizer - DSMB, Chair, DMD gene therapy trial 7) Audentes - DSMB member, MTM gene therapy trial

    Gifts:
    1. 1) Non-profit for myotonic dystrophy cognitive function in adolescents; from family benefactor 2) Non-profit for myotonic dystrophy genotype-phenotype correlations; from family benefactor

    Funding for travel or speaker honoraria:
    1. 1) Biogen, Inc. travel to Boston and Berlin for advisory meeting (2016, 2017, 2018, 2019) 2) AveXis travel to Frankfort, Chicago, Mendoza, Miami and Paris advisory meetings (2017, 2018, 2019) 3) Novartis travel to London advisory meeting (2018) 4) Spinal Muscular Atrophy Foundation, travel to Dallas, Anaheim for annual conference, (2018, 2019) 5) Genentech for presentation at AAP (2020) 6) Biogen for presentation at AANEM (2020)

    Editorial Boards:
    1. NONE

    Patents:
    1. (1) Patent on Myotonic Dystrophy type 2 genetic testing, licensed to Athena Diagnostics (2) Patent on Spinocerebellar Ataxia type 5 genetic testing, licensed to Athena Diagnostics

    Publishing Royalties:
    1. NONE

    Employment, Commercial Entity:
    1. Stanford University, Departments of Clinical Neuroscience, Professor, 9 years University of Minnesota, Department of Neurology, Professor, 19 years University of California, San Francisco, Asst. Professor, 6 years

    Consultancies:
    1. 1) Isis Pharmaceuticals – Myotonic Dystrophy Advisor 2) Biogen, Inc. – SMA Advisory Board 3) Cytokinetics, Inc. – SMA Advisor 4) Sarepta Therapeutics – Duchenne Advisor 5) PTC Therapeutics - Duchenne Advisor 6) AveXis, Inc - SMA Advisor 7) Santhera, Inc - DMD Advisor 8) Pfizer, Inc - Myotonic Dystrophy Advisor 9) Novartis, Inc. - SMA Advisor 10) Affinia Therapeutics - Gene Therapy Advisor 11) Shift Therapeutics - Genetic modification Advisor 12) Avidity Bio - myotonic dystrophy Advisor

    Speakers' Bureaus:
    1. NONE

    Other Activities:
    1. NONE

    Clinical Procedures or Imaging Studies:
    1. NONE

    Research Support, Commercial Entities:
    1. (1) Genzyme Corporation (2) Ionis Pharmaceuticals (3) Sarepta Pharmaceuticals (4) Cytokinetics, Inc. (5) AveXis, Inc. (6) Biogen, Inc. (7) Bristol-Myers. (8) Roche. (9) PTC Therapeutics. (10) Wave Therapeutics. (11) Audentes Pharmaceuticals (12) AMO Pharmaceuticals

    Research Support, Government Entities:
    1. NIH/NINDS, NS058901, Project PI, 2008-2018

    Research Support, Academic Entities:
    1. NONE

    Research Support, Foundations and Societies:
    1. (1) Muscular Dystrophy Association (2) Myotonic Dystrophy Foundation (3) Spinal Muscular Atrophy Foundation (4) CureSMA

    Stock/Stock Options/Board of Directors Compensation:
    1. NONE

    License Fee Payments, Technology or Inventions:
    1. NONE

    Royalty Payments, Technology or Inventions:
    1. (1) DM2 genetic testing, Athena Diagnostics, 2002-2020 (2) SCA5 genetic testing, Athena Diagnostics, 2007-2020

    Stock/Stock Options, Research Sponsor:
    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:
    1. NONE

    Legal Proceedings:
    1. NONE

  1. Departments of Neurology (TD, CW, SDY, KAH, JS, JWD), Rehabilitation (CEM), and Radiology (MZ, MW), Stanford University School of Medicine, Palo Alto, CA; Departments of Biostatistics and Computational Biology (MPM), and Neurology (MPM and WBM), University of Rochester Medical Center, NY; Department of Neurology (AP,BTD), Boston Children's Hospital, MA; Department of Neurology (DCD), Columbia University, New York; Department of Neurology (AMG EAK, ZZ) Children's Hospital of Philadelphia, PA; and Department of Pediatrics (RSF) Nemours Children's Hospital, Orlando, FL.
  1. Correspondence
    Dr. Duong trduong{at}stanford.edu
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