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May 21, 2019; 92 (21) ArticleOpen Access

Nusinersen in later-onset spinal muscular atrophy

Long-term results from the phase 1/2 studies

Basil T. Darras, Claudia A. Chiriboga, Susan T. Iannaccone, Kathryn J. Swoboda, Jacqueline Montes, Laurence Mignon, Shuting Xia, C. Frank Bennett, Kathie M. Bishop, Jeremy M. Shefner, Allison M. Green, Peng Sun, Ishir Bhan, Sarah Gheuens, Eugene Schneider, Wildon Farwell, Darryl C. De Vivo
First published April 24, 2019, DOI: https://doi.org/10.1212/WNL.0000000000007527
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Article Information

vol. 92 no. 21 e2492-e2506
DOI: 
https://doi.org/10.1212/WNL.0000000000007527
PubMed: 
31019106
Published By: 
Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology
Print ISSN: 
0028-3878
Online ISSN: 
1526-632X
History: 
  • Received July 12, 2018
  • Accepted in final form January 25, 2019
  • First Published April 24, 2019.

Article Versions

Copyright & Usage: 
Copyright © 2019 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. Basil T. Darras, MD,
    2. Claudia A. Chiriboga, MD, MPH,
    3. Susan T. Iannaccone, MD,
    4. Kathryn J. Swoboda, MD, FACMG,
    5. Jacqueline Montes, PT, EdD, NCS,
    6. Laurence Mignon, PhD,
    7. Shuting Xia, MS,
    8. C. Frank Bennett, PhD,
    9. Kathie M. Bishop, PhD,
    10. Jeremy M. Shefner, MD, PhD,
    11. Allison M. Green, PhD,
    12. Peng Sun, PhD,
    13. Ishir Bhan, MD, MPH,
    14. Sarah Gheuens, MD, PhD,
    15. Eugene Schneider, MD,
    16. Wildon Farwell, MD, MPH and
    17. Darryl C. De Vivo, MD
    18. on behalf of the ISIS-396443-CS2/ISIS-396443-CS12 Study Groups
  1. Basil T. Darras, MD,
    2. Scientific Advisory Boards:

    1. (1) Hoffman LaRoche (2) Cytokinetics, Inc. (3) BMS, 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. Speaker honoraria from Biogen

    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) Valerion Therapeutics (MTM) (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

  2. Claudia A. Chiriboga, MD, MPH,
    3. Scientific Advisory Boards:

    1. Advisory board member for Biogen, Avexis, Roche, Genentec

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Biogen speaker honoraria 2018

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. Biogen speaker (see above)

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. research sponsors Biogen (CS11) Avexis-Strive and Sprint Roche Pharmaceuticals (Sunfish, Jewelfish)

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. SMA foundation- natural history study Cure SMA- Spinraza staff support MDA- MDA Care Center

    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. Susan T. Iannaccone, MD,
    4. Scientific Advisory Boards:

    1. Member DSMB, Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study anaLyzing Pharmacokinetics at three dose Levels In Children and Adolescents with Assessment of Safety and Tolerability of Omigapil Santhera AveXis Ad Board Sarepta Ad Board

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. American Academy of Pediatrics, 2018

    Editorial Boards:

    1. Editorial board: Neuromuscular Disorders, Journal of Pediatric Rehabilitation Medicine

    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. Site PI for: PTC sponsored clinical trial of Ataluren in Duchenne muscular dystrophy: A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy Extension Study of Ataluren in Subjects with Nonsense- Mutation-Mediated Duchenne and Becker Muscular Dystrophy, PTC Therapeutics, 3 patients previously enrolled now in open label. 2011 - present An Open-Label, Multi-Center, 48-Week Study with a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy Sarepta 2014 - present Biogen SHINE, site coPI AveXis STRIVE, STRONG, SPRINT, site PI Capricor HOPE2, site PI

    Research Support, Government Entities:

    1. Site CoPI for NIH sponsored NeuroNEXT 2018-2023 Site CoPI for Wellstone Center for Muscular Dystrophy (NIH U54 AR068791-01 ) 2015-2020

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. Muscular Dystrophy Association, Care Center grant: coDirector Cure SMA Center of Excellence, coDirector

    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. Kathryn J. Swoboda, MD, FACMG,
    5. Scientific Advisory Boards:

    1. Cure SMA Scientific Advisory Board AHC foundation Scientific Advisory Board Biogen Scientific Advisory Board

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Biogen Ionis

    Editorial Boards:

    1. 1. Journal of Neuromuscular Disorders, Editorial Board, 2015- present

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. Biogen AveXis

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. 1. Biogen: PI for NURTURE, phase 2 trial of nusinursen in pre- symptomatic infants with SMA; April 2016-present 2. AveXis: PI for SPR1NT, phase 3 Pre-Symptomatic Study of Intravenous AVXS-101 in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2; July 2018-present

    Research Support, Government Entities:

    1. NIH RO1-HD69045 for newborn screening pilot study for SMA, PI, 2011-18, NICHD And NIH R21-NS108015, NINDS, Pathologic studies in SMA, PI, 2018-2020

    Research Support, Academic Entities:

    1. Professor, Adjunct, University of Utah Department of Neurology Lecturer, Harvard Medical School

    Research Support, Foundations and Societies:

    1. Cure SMA clinical support grant, PI, 2018-2019 Cure SMA research grant, PI, 2018-2020

    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. Jacqueline Montes, PT, EdD, NCS,
    6. Scientific Advisory Boards:

    1. (1) Roche Pharmaceuticals (2) Biogen (3)Cytokinetics (4) Scholar Rock

    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) Isis Pharmaceuticals (2) Biogen

    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. National Institute of Health: Eunice Kennedy Shriver National Institute of Child Health and Human Development (K01HD084690), Principal Imvestigator; 2016-2021

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. (1) SMA Foundation (2) Muscular Dystrophy Association Clinical Trials Travel Grant (575870).

    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. Laurence Mignon, PhD,
    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. (1) Ionis Pharmaceuticals, Inc, Director Clinical Development, 2013-2018

    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. Shuting Xia, MS,
    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. Ionis Pharmaceuticals, associate director, 18 years

    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

  8. C. Frank Bennett, PhD,
    9. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. Inventor on several issued patents covering nusinersen

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. Ionis Pharmaceuticals, Senior Vice President Research,Employed for 29 years

    Consultancies:

    1. Experimental Therapeutic Center in Singapore Stock/Stock Options, Medical Equipment & Materials: Own stock and have stock options in Ionis Pharmaceuticals

    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. Kathie M. Bishop, PhD,
    10. Scientific Advisory Boards:

    1. Unpaid scientific advisory board member for the non- profit groups the Myotonic Dystrophy Foundation and the Spinal Muscular Atrophy 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. I was a full-time employee of Isis Pharmaceuticals from September 2009 to March 2015. Currently a full-time employee of Otonomy Inc.

    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. Jeremy M. Shefner, MD, PhD,
    11. Scientific Advisory Boards:

    1. I served on a Scientific Advisory Boards sponsored by Biogen-Idec, AveXis, Orphazyme, Brainstorm and Cytokinetics related to new ALS therapies. I am also the Medical Monitor for the Braingate Brain-Computer Interface Program.

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. non-profit: I received an honorarium for giving Grand Rounds Massachusetts General Hospital.

    Editorial Boards:

    1. UpToDate, Neuromuscular Section Editor, 2007-present. Editorial Board, Neurotherapeutics and the ALS Journal.

    Patents:

    1. NONE

    Publishing Royalties:

    1. UpToDate, Neuromuscular Section Editor, 2007-present.

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. Commercial: I have consulted for Cytokinetics, and Biohaven.

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. Biogen Idec, clinical trial management. Cytokinetics, clinical trial management and site investigator. Amylyx, clinical trial management Brainstorm, clinical trial management

    Research Support, Government Entities:

    1. NIH through subcontracts from the Massachusetts General Hospital, trial management. NIH: 1U24NS107204-01, Principal Investigator, 2018-2013

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. ALS Association,principal investigator of TREAT ALS grant and experimental markers of ALS progression grant. ALS Finding a Cure 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

  11. Allison M. Green, 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. Excel Scientific Solutions, Envision Pharma Group, medical writer, 2 years; Biogen provided funding for medical writing support in the development of this report to Excel Scientific Solutions.

    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. Peng Sun, PhD,
    13. 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. Biogen, Director of Biostatistics, 4 years

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. Biogen as an employee

    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. Biogen, 4 years

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  13. Ishir Bhan, MD, MPH,
    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. I am an employee of Biogen (2 years)

    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. Biogen (stock)

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  14. Sarah Gheuens, MD, PhD,
    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. Biogen, senior medical director, 6 years

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other activities:

    1. I am an employee of Biogen and hold stock.

    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. Eugene Schneider, MD,
    16. 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. Ionis Pharmaceuticals, VP Clinical Development, 2013-present

    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. Ionis Pharmaceuticals - employee and stock-holder

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  16. Wildon Farwell, MD, MPH and
    17. 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. I am an employee of Biogen. My employment with Biogen begn in 2010.

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. I am an employee of Biogen.

    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. Darryl C. De Vivo, MD
    18. Scientific Advisory Boards:

    1. SMA Foundation. The Hope for Children Research Foundation. Pediatric Neurotransmitter Disease Association. International Reye Syndrome Foundation. The Will Foundation Chairman, Data Safety Monitoring Board, Cytokinetics Chairman, Canavan Disease Foundation Advisory Board, NYC Scientific Advisory Board, Metafora Glut1 Deficiency Foundation The Grace Science Foundation

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Speaker Honorarium from the American Academy of Neurology

    Editorial Boards:

    1. Current Opinion in Neurology, Editorial Board Medlink in Neurology, Editorial Board Journal of Pediatric Neurology Editorial Board (Turkish journal)

    Patents:

    1. Gene therapy for Glut1 Deficiency patent International PCT Patent Application No. PCT/US2016/021810 Based on U.S. Provisional Patent Application Serial No.: 62/130,899 Title: RECOMBINANT GLUT1 ADENO-ASSOCIATED VIRAL VECTOR CONSTRUCTS AND RELATED METHODS FOR RESTORING GLUT1 EXPRESSION Filed: March 10, 2016 Our Ref.: 01001/003887-WO0

    Publishing Royalties:

    1. Elsevier Publishers. Lippincott Publishers.

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. Ionis Pharmaceuticals Biogen Pharmaceuticals Ultragenyx Pharmaceuticals

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. Ionis Pharmaceuticals Biogen Pharmaceuticals Ultragenyx Pharmaceuticals Sarepta Therapeutics PTC Therapeutics

    Research Support, Government Entities:

    1. NIH, DOD

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. SMA Foundation. The Hope for Children Research Foundation. The Will Foundation The Pediatric Neurotransmitter Disease Association The 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. Expert witness.

  1. From the Department of Neurology (B.T.D.), Boston Children's Hospital, MA; Departments of Neurology (C.A.C., J.M., D.C.D.), Pediatrics (C.A.C., D.C.D.), and Rehabilitation and Regenerative Medicine (J.M.), Columbia University Irving Medical Center, New York, NY; Department of Pediatrics (S.T.I.), University of Texas Southwestern Medical Center, Dallas; Department of Neurology (K.J.S.), Massachusetts General Hospital, Boston; Ionis Pharmaceuticals, Inc. (L.M., S.X., C.F.B., E.S.), Carlsbad, CA; employee of Ionis Pharmaceuticals, Inc. (K.M.B.), Carlsbad, CA, during design and conduct of this study, current employee of Otonomy, San Diego, CA; Department of Neurology (J.M.S.), Barrow Neurologic Institute, Phoenix, AZ; Excel Scientific Solutions (A.M.G.), Southport, CT; and Biogen (P.S., I.B., S.G., W.F.), Cambridge, MA.
  1. Correspondence
    Dr. Darras basil.darras{at}childrens.harvard.edu
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