Precious SMA natural history data
A benchmark to measure future treatment successes
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Article Information
- First Published July 25, 2018.
Article Versions
- Previous version (July 25, 2018 - 12:45).
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Author Disclosures
- Basil T. Darras, MD and
- Darryl C. De Vivo, MD
- Basil T. Darras, MD and
(1) Hoffman LaRoche (2) Cytokinetics, Inc. (3) Audentes, Inc (4) Sarepta Therapeutics (5) Biogen (6) PTC Therapeutics (7) AveXis
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Speaker honoraria from Biogen
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(1) Various titles, UpToDate, 1999-present (2) 2 videotaped presentations,
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(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.
Biogen
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(1) PTC Therapeutics (2) Valerion Therapeutics (MTM) (3) Ionis Pharmaceuticals (4) Sarepta Therapeutics (5) Biogen (6) Summit (7) AveXis (8) Roche
(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.
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(1) SMA Foundation (2) Muscular Dystrophy Association (3) Slaney Family Fund for SMA
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- Darryl C. De Vivo, MD
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
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Speaker Honorarium from the American Academy of Neurology
Current Opinion in Neurology, Editorial Board Medlink in Neurology, Editorial Board Journal of Pediatric Neurology Editorial Board (Turkish journal)
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
Elsevier Publishers. Lippincott Publishers.
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Ionis Pharmaceuticals Biogen Pharmaceuticals Ultragenyx Pharmaceuticals
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Ionis Pharmaceuticals Biogen Pharmaceuticals Ultragenyx Pharmaceuticals Sarepta Therapeutics
NIH, DOD
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SMA Foundation. The Colleen Giblin Foundation. The Will Foundation The Pediatric Neurotransmitter Disease Association The Glut1 Deficiency Foundation
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Expert witness.
- 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.
- Correspondence
Dr. Darras basil.darras{at}childrens.harvard.edu
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