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August 21, 2018; 91 (8) Editorial

Precious SMA natural history data

A benchmark to measure future treatment successes

Basil T. Darras, Darryl C. De Vivo
First published July 25, 2018, DOI: https://doi.org/10.1212/WNL.0000000000006026
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Article Information

vol. 91 no. 8 337-339
DOI: 
https://doi.org/10.1212/WNL.0000000000006026
PubMed: 
30045956
Published By: 
Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology
Print ISSN: 
0028-3878
Online ISSN: 
1526-632X
History: 
  • First Published July 25, 2018.

Article Versions

Copyright & Usage: 
© 2018 American Academy of Neurology

Author Disclosures

    1. Basil T. Darras, MD and
    2. Darryl C. De Vivo, MD
  1. Basil T. Darras, MD and
    2. Scientific Advisory Boards:

    1. (1) Hoffman LaRoche (2) Cytokinetics, Inc. (3) Audentes, Inc (4) Sarepta Therapeutics (5) Biogen (6) PTC Therapeutics (7) AveXis

    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) 2 videotaped presentations,

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. (1) Leerink Partners, LLC (2) Cowen and Company (3) Guidepoint Global Consultation (4) Vertex Pharma (5) Clearview Healthcare Partners (6) Gershon Lehrman Group (7) Putnam Associates (8) Wallachbeth Capital, LLC (9) Frankel Group (10) Voyager Therapeutics (11) Noble Insights (12) Jefferies and Company (13) Krog & Partners, Inc.

    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) Ionis Pharmaceuticals (4) Sarepta Therapeutics (5) Biogen (6) Summit (7) AveXis (8) Roche

    Research Support, Government Entities:

    1. (1) NIH/NIAMS, 2P01 NS040828-6A11, Co-PI, 2000-2012. (2) NIH/NINDS, 1U10NS077269, Site-PI/PD, 2011-2018. (3) NIH/NIAMS, 1R01AR060850, Co-PI, 2011-2015. (4) NIH/NINDS, 5U01NS061799, FOR-DMD, Site-PI, 2012-2016. (5) 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. Darryl C. De Vivo, MD
    3. 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 Wilsey 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

    Research Support, Government Entities:

    1. NIH, DOD

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. SMA Foundation. The Colleen Giblin 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.), Neuromuscular Program, Boston Children's Hospital, and Harvard Medical School, Boston, MA; and Pediatric Neurology Service at Columbia University Irving Medical Center (D.C.D.), New York, NY.
  1. Correspondence
    Dr. Darras basil.darras{at}childrens.harvard.edu
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