Nusinersen Treatment in Adults With Spinal Muscular Atrophy
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Article Information
- 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.
Author Disclosures
- Tina Duong, PT, PhD,
- Connie Wolford, MSN, FNP-BC (cwolford{at}stanfordhealthcare.org),
- Michael P. McDermott, PhD (michael_mcdermott{at}urmc.rochester.edu),
- Chelsea E. Macpherson, PT, DPT, NCS (cem2183{at}gmail.com),
- Amy Pasternak, PT, DPT, PCS (amy.pasternak{at}childrens.harvard.edu),
- Allan M. Glanzman, PT, DPT, PCS (glanzmana{at}email.chop.edu),
- William B. Martens, BA (bill_martens{at}urmc.rochester.edu),
- Elizabeth Kichula, MD, BA (kichulae{at}email.chop.edu),
- Basil T. Darras, MD (basil.darras{at}childrens.harvard.edu),
- Darryl C. De Vivo, MD (dcd1{at}cumc.columbia.edu),
- Zarazuela Zolkipli-Cunningham, MBChB, MRCP (zolkipliz{at}email.chop.edu),
- Richard S. Finkel, MD (richard.finkel{at}stjude.org),
- Michael Zeineh, MD, PhD,
- Max Wintermark, MD (mzeineh{at}stanford.edu),
- Jacinda Sampson, MD, PhD (jacindas{at}stanford.edu),
- Katharine A. Hagerman, PhD (khagerma{at}stanford.edu),
- Sally Dunaway Young, PT, DPT (sdy{at}stanford.edu) and
- John W. Day, MD, PhD on behalf of the Stanford Neuromuscular Coordinating Center (jwday{at}stanford.edu)
- Tina Duong, PT, PhD,
Advisory board or consultancy for 1) Commercial: Roche, Novartis, Biogen, Scholar Rock 2) Non-Profit: SMA foundation
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1) Commercial: Biogen, Roche, Genentech, Trinds, ATOM International
1)Commercial: Genentech, Roche, Biogen 2) CureSMA
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1) SMA Foundation
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- Connie Wolford, MSN, FNP-BC (cwolford{at}stanfordhealthcare.org),
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Biogen speaker's bureau
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- Michael P. McDermott, PhD (michael_mcdermott{at}urmc.rochester.edu),
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.
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Movement Disorders, Editorial Board, 2010-2020; Chance, Editor, 2014-2019.
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Received consulting fees from Fulcrum Therapeutics, Inc.
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Received grant support from PTC Therapeutics.
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.
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Received grant support from the Spinal Muscular Atrophy Foundation, Cure SMA, and the Parkinson Study Group.
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- Chelsea E. Macpherson, PT, DPT, NCS (cem2183{at}gmail.com),
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- Amy Pasternak, PT, DPT, PCS (amy.pasternak{at}childrens.harvard.edu),
1. Scholar Rock 2. Biogen
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1. Biogen 2. Stanford CME
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1. Biogen
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- Allan M. Glanzman, PT, DPT, PCS (glanzmana{at}email.chop.edu),
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Audentis pharmaceutical, atom international, scholar rock, biogen pharmaceutical, avexis pharmaceutical
Editorial Board, Physical & Occupational Therapy in Pediatrics 2017-present
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audentes pharmaceutical, aspa pharmaceutical, biogen pharmaceutical, avexis pharmaceutical, roche pharmaceutical
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Adjunct lecturer Arcadia University
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CHOP INTEND royalty through employer
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- William B. Martens, BA (bill_martens{at}urmc.rochester.edu),
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- Elizabeth Kichula, MD, BA (kichulae{at}email.chop.edu),
(1) PTC Therapeutics (2) Biogen (3) Stealth BioTherapeutics (4) Avexis
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Natera
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Genentech
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- Basil T. Darras, MD (basil.darras{at}childrens.harvard.edu),
(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
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(1) Various titles, UpToDate, 1999-present (2) One videotaped presentation,
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Biogen
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(1) PTC Therapeutics (2) Scholar Rock (3) Sarepta Therapeutics (4) Biogen (5) Summit (6) AveXis (7) Roche (8) Fibrogen (9) Santhera (10) Cytokinetics
(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.
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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 (dcd1{at}cumc.columbia.edu),
Biogen DEVOTE Steering Committee; AveXis RESTORE Steering Committee; ASPA Therapeutics DSMC; Roche (Genentech) Advisory Board; Scholar Rock Advisory Committee.
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2020 Distinquished Lecture, Mount Sinai Medical Center, New York
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Glut1DS gene therapy
Neuromuscular Disorders in Infancy, Childhood and Adolescence, second edition, 2015 Academic Press
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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.
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Principal Investigator for Clinical Trials funded by Biogen, Sarepta, PTC, Ultragenyx, Scholar Rock, GliaPharm, Fulcrum Therapeutics and performed at Columbia University.
DoD Program Project 2020-2024 focusing on treatments for mitochondrial diseases.
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Hope for Children Research Foundation; Glut1 Deficiency Foundation;
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- Zarazuela Zolkipli-Cunningham, MBChB, MRCP (zolkipliz{at}email.chop.edu),
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1. Oxygen Nanosensor to measure tissue Oxygen levels. 1065-18. 2. Methods of Treating Mitochondrial Dysfunction. 62/689.
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NICHD U24 Leigh syndrome gene curation grant support.
Pediatric Neuromuscular Clinical Research Network support from the Spinal Muscular Atrophy Foundation (SMAF)and CureSMA.
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- Richard S. Finkel, MD (richard.finkel{at}stjude.org),
(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
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(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
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 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.
Swaiman's Principles and Practice of Pediatric Neurology, 6th edition; Elsevier, 2017, royalties received 2018-20
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(1) Capricor: 2017-19 (2) Biogen: 2014-20 (3) Roche: 2015-20 (4) AveXis: 2015-20 (5) Novartis: 2015-20
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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.
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
(1) 5U01NS061799, Co-I, 2010-19 (2) RO1AR056973, Co-I, 2010-19 (3) 1U54NS065712-01, Co-I, 2009-19
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(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
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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.
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- Michael Zeineh, MD, PhD,
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(1) consultant for Biogen.
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(1) GE Healthcare
(1) NIH/NIA, 1R01AG061120-01, (PI Zeineh), 3/2019 Â 12/2023
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- Max Wintermark, MD (mzeineh{at}stanford.edu),
(1) Commercial entity: Nines; (2) Commercial entity: Icometrix; (3) Commercial entity: Magnetic Insight; (4) Commercial entity: MoreHealth; (5) Commercial entity: Subtle Medical
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- Jacinda Sampson, MD, PhD (jacindas{at}stanford.edu),
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)
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Funding for travel to MDF 2019 annual meeting
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Expert testimony, not related to industry
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Marigold Foundation (Myotonic dystrophy study).
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- Katharine A. Hagerman, PhD (khagerma{at}stanford.edu),
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(1) Biogen Inc
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(1) SMA Foundation, (2) Marigold Foundation
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- Sally Dunaway Young, PT, DPT (sdy{at}stanford.edu) and
(1) Biogen (2) Roche/Genentech (3) Scholar Rock
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(1) Roche/Genentech
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(1) Biogen (2) Cure SMA (3) Scholar Rock (4) Roche/Genentech
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(1) Biogen
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(1) SMA Foundation (2) Cure SMA
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- John W. Day, MD, PhD on behalf of the Stanford Neuromuscular Coordinating Center (jwday{at}stanford.edu)
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
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
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)
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(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
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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
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
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(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
NIH/NINDS, NS058901, Project PI, 2008-2018
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(1) Muscular Dystrophy Association (2) Myotonic Dystrophy Foundation (3) Spinal Muscular Atrophy Foundation (4) CureSMA
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(1) DM2 genetic testing, Athena Diagnostics, 2002-2020 (2) SCA5 genetic testing, Athena Diagnostics, 2007-2020
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- 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.
- Correspondence
Dr. Duong trduong{at}stanford.edu
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