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September 24, 2019; 93 (13) ArticleOpen Access

Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function

Eric P. Hoffman, Benjamin D. Schwartz, Laurel J. Mengle-Gaw, Edward C. Smith, Diana Castro, Jean K. Mah, Craig M. McDonald, Nancy L. Kuntz, Richard S. Finkel, Michela Guglieri, Katharine Bushby, Mar Tulinius, Yoram Nevo, Monique M. Ryan, Richard Webster, Andrea L. Smith, Lauren P. Morgenroth, Adrienne Arrieta, Maya Shimony, Catherine Siener, Mark Jaros, Phil Shale, John M. McCall, Kanneboyina Nagaraju, John van den Anker, Laurie S. Conklin, Avital Cnaan, Heather Gordish-Dressman, Jesse M. Damsker, Paula R. Clemens, the Cooperative International Neuromuscular Research Group
First published August 26, 2019, DOI: https://doi.org/10.1212/WNL.0000000000008168
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Article Information

vol. 93 no. 13 e1312-e1323
DOI: 
https://doi.org/10.1212/WNL.0000000000008168
PubMed: 
31451516
Published By: 
Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology
Print ISSN: 
0028-3878
Online ISSN: 
1526-632X
History: 
  • Received October 26, 2018
  • Accepted in final form May 29, 2019
  • First Published August 26, 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. Eric P. Hoffman, PhD,
    2. Benjamin D. Schwartz, MD,
    3. Laurel J. Mengle-Gaw, PhD,
    4. Edward C. Smith, MD,
    5. Diana Castro, MD,
    6. Jean K. Mah, MD,
    7. Craig M. McDonald, MD,
    8. Nancy L. Kuntz, MD,
    9. Richard S. Finkel, MD,
    10. Michela Guglieri, MD,
    11. Katharine Bushby, MD,
    12. Mar Tulinius, MD,
    13. Yoram Nevo, MD,
    14. Monique M. Ryan, MD,
    15. Richard Webster, MD,
    16. Andrea L. Smith, MS,
    17. Lauren P. Morgenroth, MS,
    18. Adrienne Arrieta, MS,
    19. Maya Shimony, MPH,
    20. Catherine Siener, PT,
    21. Mark Jaros, PhD,
    22. Phil Shale, PhD,
    23. John M. McCall, PhD,
    24. Kanneboyina Nagaraju, PhD, DVM,
    25. John van den Anker, MD, PhD,
    26. Laurie S. Conklin, MD,
    27. Avital Cnaan, PhD,
    28. Heather Gordish-Dressman, PhD,
    29. Jesse M. Damsker, PhD,
    30. Paula R. Clemens, MD;
    31. the Cooperative International Neuromuscular Research Group
  1. Eric P. Hoffman, PhD,
    2. Scientific Advisory Boards:

    1. (1) Non-profit entity. Scientific Advisor, Foundation to Eradicate Duchenne (www.duchennemd.org) (2) Non-profit entity. Scientific Advisory Board, CureDuchenne Foundation (www.cureduchenne.org) (3) Non-profit entity. Scientific Advisory Board, C3 Foundation (www.curecalpain3.org) (4) Non-profit entity. Executive Committee, TREAT-NMD (www.treat-nmd.eu) (5) Non-profit entity. Advisory Board, Save Our Sons (www.saveoursons.org.au) (6) Non-profit entity. Advisory Board, Duchenne UK Foundation (www.duchenneuk.org)

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. 1. Editorial Board, European Journal of Human Genetics 2. Advisory Board, Science Translational Medicine (AAAS)

    Patents:

    1. WO 2009155056 Patent Family (VLD0001 Series). Method of treatment ? Subject matter - Methods of using VBP15 and related delta 9,11 steroid compounds for the treatment of 160+ diseases including muscular dystrophy (including Duchenne and Becker), rheumatoid arthritis, asthma, renal disease, inflammatory bowel disease, colitis, Crohn's disease, and multiple sclerosis. As data supporting disease application is acquired, divisionals that cover that disease are filed. Thus, the methods umbrella can expand with time. ? Publication Information 1. Priority Date: 5/28/2008 2. US and PCT Filing Date: 5/28/2009 3. PCT Publication Date: 12/23/2009 4. US Publication Date: 4/8/2010 5. US Publication No.: US 20100087408 US Prosecution ? Issued US Patent No. US 8,207,151. Arthritis 1. Issue Date: 6/26/2012 2. Priority date: 528/2008 3. Expiration Date: 7/9/2030, plus up to five years of potential Hatch-Waxman exclusivity 4. CLAIMS  A method of reducing the symptoms of arthritis comprising the administration, to a patient in need thereof, of a therapeutically effective amount of a compound having the structural formula of vamorolone ? Issued US Patent No. US 8,334,279. Methods of treating muscular dystrophy using VBP15 1. Issue Date: 12/18/2012 2. Priority date: 528/2008 3. Expiration Date: 5/28/2029, plus up to five years of potential Hatch-Waxman exclusivity 4. Claims: muscular dystrophy is selected from the group consisting of Duchenne muscular dystrophy, Becker muscular dystrophy, limb girdle muscular dystrophy, congenital muscular dystrophy, facioscapulohumeral muscular dystrophy, myotonic muscular dystrophy, oculopharyngeal muscular dystrophy, distal muscular dystrophy, and Emery-Dreifuss muscular dystrophy ? Issued US Patent No.: US 8,673,887. Methods of treating Crohn's disease, colitis, and inflammatory bowel disease using VBP15 o Issue Date: 3/18/2014 o Priority date: 528/2008 o Expiration Date: 5/28/2029, plus up to five years of potential Hatch-Waxman exclusivity o Claims: method of treating or reducing the symptoms of a disease selected from the group consisting of Crohn's disease, colitis, and inflammatory bowel disease, comprising the administration, to a patient in need thereof, of a therapeutically effective amount of a compound having the structural formula of vamorolone ? Issued US Patent No.: US 9,434,758. treatment of MS o Issue date: 9/5/2016 o Priority date: 5/28/2008 o Expiration Date: 5/28/2029, plus up to five years of potential Hatch-Waxman exclusivity o Claims: method of treating multiple sclerosis comprising the administration, to a patient in need thereof, of a therapeutically effective amount of a compound having the structural formula of vamorolone ? Issued US Patent No.: US 9,649,320. treatment of COPD and asthma o Issue date: 516/2017 o Priority date: 5/28/2008 o Expiration Date: 5/28/2029, plus up to five years of potential Hatch-Waxman exclusivity o Claims: method of treating or reducing the symptoms of a disease selected from the group consisting of asthma and chronic obstructive pulmonary disease, comprising the administration, to a patient in need thereof, of a therapeutically effective amount of a compound having the structural formula of vamorolone o This case is supported by US Patent 9,188,581 (11/17/2015, Eric Hoffman and Robert Freishtat) that covers methods for diagnosing and treating asthma. ex-US Filings ? National phase filings in Australia, Brazil, Canada, China, Europe, India, Japan, Mexico, New Zealand, South Africa, and South Korea and Hong Kong Australia  Issued Patent: 2009260471 granted on March 26, 2015  Expiration Date: 5/28/2029  Claims one structure for use in treating muscular dystrophy. Canada  Response to Office Action filed Nov. 15.  Claims directed to muscular dystrophy, asthma, Crohn?s disease, colitis and inflammatory bowel disease. Claims reinstated July 2017: Duchenne?s, asthma, Crohn?s disease, arthritis, colitis and inflammatory bowel disease. The application was allowed once but we have added additional use claims this time around and are hopeful to get a quick allowance.  Claims allowed include all major disease. Canadian application 2,725,008 granted Aug 2017. Very broad coverage China  Issued Patent: CN 200980124859.0 VLD0001-504- CN has been granted, and it claims thirteen steroid structures for use in treating muscular dystrophy. It also has a dependent claim to the use of the following compound in treating muscular dystrophy. Issue Date: 11/13/2013. Claims: Methods of treating muscular dystrophy  May 16 2017. VLD0001-504D2-CN. Granted chronic obstructive pulmonary disease, inflammatory bowel disease, crohn?s disease, colitis, amyotropic lateral sclerosis, multiple sclerosis, traumatic brain injury, and spinal cord injury. Issue date June 9, 2017.  Divisional application filed with claims directed towards additional diseases. VLD0001-504D1-CN was in prosecution, claiming thirteen steroid structures for use in treating arthritis, asthma, chronic obstructive pulmonary disease, inflammatory bowel disease, Crohn's disease, colitis, amyotrophic lateral sclerosis, multiple sclerosis, traumatic brain injury, and spinal cord injury. It also has a dependent claim to the use of the VBP15 above in treating those diseases. In August 2017 we decided to abandon. Japan.  Crohn?s, colitis, inflammatory bowel disease, multiple sclerosis. Patent No. 6204408. Term is 20 years with expiration 5/28/2029muscular dystrophy, asthma, chronic obstructive pulmonary disease, arthritis, traumatic brain injury, spinal cord injury, amyotropic lateral sclerosis, and Sjogren?s syndrome. Issued Patent: 5780521 granted on July 24, 2015, Expiration Date: 5/28/2029 Europe. Summary: We selected the Tier 1 and 2 countries for validation. We have selected the following countries for divisionals: Austria, Belgium, Czech Republic, Denmark, France, Germany, Hungary, Ireland, Italy, Norway, Spain, Sweden, Switzerland, The Netherlands, and the United Kingdom.  Original application claims were directed towards methods of treating muscular dystrophy, asthma, and arthritis.  Divisional application: claims are directed towards methods of treating multiple sclerosis, IBD, colitis, and Crohn's disease.  Granted EP2278978 on 9/2/2015 for asthma and arthritis ? Claims: A compound for use in treating the symptoms of a disease selected from the group consisting of arthritis and asthma, wherein said compound has the structural formula of vamorolone  Granted EP2444092 on 9/2/2015 for treating Crohn?s, colitis and IBD ? Claims: A compound for use in a method of treatment of a disease selected from the group consisting of Crohn's disease, colitis, inflammatory bowel disease, and multiple sclerosis, wherein said compound has the structural formula of vamorolone  Pending EP14173342.8, September 2017. Allowed 10/22/2018 as EP2805720 ? claims to vamorolone and VBP6 in the treatment of muscular dystrophy and subtypes, including Duchenne. Claims I ncludemuscular dystrophy is selected from the group consisting of Duchenne muscular dystrophy, Becker muscular dystrophy, limb girdle muscular dystrophy, congenital muscular dystrophy, facioscapulohumeral muscular dystrophy, myotonic muscular dystrophy, oculopharyngeal muscular dystrophy, distal muscular dystrophy, and Emery-Dreifuss muscular dystrophy  Regarding the issue of broadening claims in Europe, the claims we currently have presented are identical in chemical scope to claims that were present in the PCT case as filed. Our European attorney has indicated that he does not think there will be an issue with the claim scope. Mexico  Issued Patent: 326125 was granted on December 9, 2014  Expiration Date: 5/28/2029  Claims one structure for use in treating muscular dystrophy, arthritis, asthma, and COPD New Zealand  Issued Patent: NZ 589444  Expiration Date: 5/28/2029  Claims: Methods of treating arthritis using VBP15 and other compounds  Divisional application granted on July 1, 2014  Divisional claims are directed towards methods of treating broad list of diseases. South Africa  Issued Patent: ZA 2010/08256  Expiration Date: 5/28/2029  Claims: Methods of treating broad list of diseases South Korea  Claims to muscular dystrophies and arthritis allowed (VLD0001-511-KR); Issue fee due on January 28, 2016  Asthma allowed (divisional application 1) with general muscular dystrophy  Claims to Crohn?s, IBD and ulcerative colitis with fee due on June 14, 2017. India  VLD0001-506-IN: Application pending. Evidence was submitted for oral hearing on November 24, 2017. Brazil  Applications pending, substantive prosecution has not yet begun WO 2011127048 Patent Family (VLD0002 Series). Composition of matter ? Subject matter - covers additional delta 9,11 steroid compounds for the treatment of the diseases disclosed in WO '056. ? Publication Information 1. Priority Date: 4/5/2010 2. US and PCT Filing Date: 4/5/2011 3. PCT Publication Date: 10/13/2011 4. US Publication Date: 8/1/2013 5. US Publication No.: US 20130196962 6. US 9,198,921 issued on 12/1/2015 7. Expiration Date: 4/5/2031 ? US 9,198,921 (12/1/2015) issued 1. This links to application 13/630,650 and corresponds to PCT/US2011/031238 2. Continuation application filed on November 12, 2015 ? ex-US Filings 1. No national phase applications are pending WO 2013082253 Patent Family (VLD0003 Series) ? Subject matter - covers the treatment of additional diseases using VBP15 and the additional delta 9,11 steroid compounds disclosed in WO '056 and WO '048. ? Publication Information 1. Priority Date: 11/29/2011 2. US and PCT Filing Date: 11/29/2012 3. PCT Publication Date: 6/6/2013 ? Currently pending U.S. application 14/360384; U.S. publication 2015-0011519 (application, corresponds to WO2013/082253). No issued patents. ? ex-US Filings 1. Pending applications filed in Australia, Canada and Europe. Substantive prosecution has not yet begun. VLD0006 Series. Decided not to nationalize and to pursue VLD0006-201-US (Oct 2017) ? Filed 6/29/2015 Provisional 62/186214 ? Non provisional June 29, 2016. US Application No.: 15/197,118, PCT Application No.: PCT/US2016/040098 ? Publication Date 12/29/2016 ? PCT application published on January 5, 2017 as Publication No. WO2017/004205. ? National phase filing 30-month date 12/29/2017 ? Method claims that cover new diseases (see below)

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. (1). Commercial entity. ReveraGen BioPharma. Co-founder, CEO, President 2013-present (2). Commercial entity. AGADA BioSciences. Co-founder, Vice President, 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. (1) Commercial entity. TRiNDS LLC. Distributions as partial owner.

    Research Support, Government Entities:

    1. Ongoing Research Support 1 R43 AR073547 01 DEFINITION OF PHARMACODYNAMIC BIOMARKERS FOR JUVENILE DERMATOMYOSITIS FOR CLINICAL TRIALS HOFFMAN, ERIC P. et al. REVERAGEN BIOPHARMA, INC. 2018 NIAMS NIAMS $225,000 5 U54 HD090257 03 8635 GENOMICS ​AND ​PROTEOMIC-CORE HOFFMAN, ERIC P. CHILDREN'S RESEARCH INSTITUTE 2018 NICHD $124,781 2 R44 NS095423 03 PIVOTAL TRIAL OF VAMOROLONE IN DUCHENNE MUSCULAR DYSTROPHY HOFFMAN, ERIC P. et al. REVERAGEN BIOPHARMA, INC. 2018 NINDS NINDS $1,500,000

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. (1). Muscular Dystrophy Association (2). Save Our Sons (3). Parent Project Muscular Dystrophy (4). CureDuchenne (5). DuchenneAlliance (6). DuchenneUK (7). Foundation to Eradicate Duchenne

    Stock/Stock Options/Board of Directors Compensation:

    1. (1). Commercial entity. ReveraGen Biopharma. Co-Founder, founder shares. (2). Commercial entity. AGADA BioSciences. Co-founder, founder shares (3). Commercial entity. TRiNDS LLC. Co-founder, founder shares

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. (1). Commercial entity. ReveraGen Biopharma. Co-Founder, founder shares. 2013-present (2). Commercial entity. AGADA BioSciences. Co-founder, founder shares. 2013-present (3). Commercial entity. TRiNDS LLC. Co-founder, founder shares. 2016-present

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  2. Benjamin D. Schwartz, MD,
    3. 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) ReveraGen (2) Hospira (3) Pfizer (4) Baxter Healthcare (5) Horizon (6) TRINDS (7) Cytometix (8) Xenamed

    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

  3. Laurel J. Mengle-Gaw, PhD,
    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. (1) Pfizer, (2) Hospira, (3) Baxter Healthcare, (4) Horizon, (5) Cytometix, (6) Xenamed, (7) ReveraGen BioPharma, (8) TRiNDS

    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

  4. Edward C. Smith, MD,
    5. Scientific Advisory Boards:

    1. DSMB Member Phase I/IIa gene transfer clinical trial for Duchenne Muscular Dystrophy using rAAVrh74.MCK.GALGT2 Kevin M. Flanigan, M.D., Principal Investigator NIAMS Grant # R21 AR068040

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Travel and speaker honoraria for consulting services: Biogen, Avexis

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. Commercial (Biogen)

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. Salary support for PI role on Pompe clinical trial sponsored by NIH/NIAMS U01-AR07-1693

    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. Diana Castro, MD,
    6. Scientific Advisory Boards:

    1. (1) Avexis- Commercial (2) Genetech- Commercial (3) Biogen- Commercial

    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. (1) Biogen- Commercial

    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

  6. Jean K. Mah, MD,
    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. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. Research grants as site investigator for clinical trials, unrelated to the present study: PTC Therapeutics Bristol Myers Squibb Italfarmaco S.p.A ReveraGen Pharma NS Pharma Pfizer Inc Cytokinetics Inc. Biogen Roche Sanofi-Genzyme Sarepta Novartis Inc.

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. FSH Society Grant# FSHS-02010-04 Muscular Dystrophy Canada FSHD Global Research 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

  7. Craig M. McDonald, MD,
    8. Scientific Advisory Boards:

    1. (1) PTC Therapeutics, scientific advisory board concerning Duchenne muscular dystrophy, (2) Eli Lilly, scientific advisory committee concerning Duchenne muscular dystrophy, (3) Catabasis, scientific advisory Board, (4) Sarepta Therapeutics, scientific advisory board, Capricor Scientific Advisory Board, (5) Santhera Pharmaceuticals Scientific Advisory Board, (6) Astellas Scientific Advisory Board

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. (1) PTC Therapeutics, speaker honorarium for Duchenne muscular dystrophy educational symposium, (2) Marathon, speaker honorarium for Duchenne muscular dystrophy educational symposium, (3) Catabasis, honoraria for educational symposium, (4) Sarepta Therapeutics, honoraria for educational symposium, (5) Capricor, honoraria for educational symposium, (6) Santhera Pharmaceuticals, honoraria for educational symposium

    Editorial Boards:

    1. Editorial Advisory Board and Associate Editor, Muscle & Nerve; Editorial Advisory Board Neuromuscular Disorders

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. (1) PTC Therapeutics, consulting concerning Duchenne muscular dystrophy, (2) Catabasis, consulting concerning Duchenne muscular dystrophy, (3) Sarepta Therapeutics, consulting concerning Duchenne muscular dystrophy, (4) Capricor, consulting Duchenne muscular dystrophy, (5) Santhera Pharmaceuticals, consulting concerning Duchenne muscular dystrophy (6) Astellas consulting concerning Duchenne muscular dystrophy, (7) Cytokinetics, consulting concerning SMA, (8) Italfarmaco consulting concerning Duchenne muscular dystrophy

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. (1) Sarepta Therapeutics, (2) PTC Therapeutics, (3) Pfizer, (4) Reveragen, (5) Italfarmaco, (6) Capricor, (7) Roche, (8) Catabasis, (9) Santhera Pharmacuricals

    Research Support, Government Entities:

    1. National Institute of Disability and Rehabilitation Research Grant #H133B090001; Rehabilitation Research and Training Center in Neuromuscular Diseases: Enhancing Health and Wellness of Individuals with Neuromuscular Diseases. October 1, 2009 to September 30, 2015 - $4,000,000. Principal Investigator: Craig M. McDonald National Institutes of Arthritis, Musculoskeletal and Skin Diseases Research (NIAMS), Grant #1R01AR061875: Clinically Meaningful Outcomes for Duchenne Muscular Dystrophy Therapeutic Trials. September 1, 2011 to August 31, 2015 - $1,320,122 Principal Investigator: Craig M. McDonald, MD National Institutes of Arthritis, Musculoskeletal and Skin Diseases Research (NIAMS), Grant # 5R01AR062380: Biomarker discovery and validation in a Duchenne dystrophy natural history study. September 1, 2011 to August 31, 2015 - $1,318,122 Principal Investigator: Craig M. McDonald, MD Department of Defense Congressionally Directed Medical Research Program (CDMRP) Grant # MD130062; Grants.gov ID Number: GRANT11522309 Development of a lifespan-based novel composite person- reported outcome measure using data from the CINRG Duchenne natural history study. July 1, 2014 to June 30 2017, $1,001,162 Principal Investigator: Craig M. McDonald, MD Department of Defense Congressionally Directed Medical Research Program (CDMRP) Grant # MD160094; Grants.gov ID Number: GRANT12274832 Validation of novel community-based measures of physical activity in 2.5 year old children with Duchenne muscular dystrophy. July 1, 2017 to June 30 2020, $942,000 Principal Investigator: Craig M. McDonald, MD

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. (1) Muscular Dustrophy Association, (2) Parent Project Muscular Dystrophy

    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. Nancy L. Kuntz, MD,
    9. Scientific Advisory Boards:

    1. Dr. Kuntz has served on scientific advisory boards for AveXis, Audentes, BioGen Idec, Sarepta Pharmaceuticals, Marathon Pharmaceuticals and Cytokinetics.

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Biogen, speaker Honoraria for lectures in Australia and Japan;

    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. (1) Cooperative International Neuromuscular Research Group/DOD and NIH, site investigator, 2010-present

    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. Richard S. Finkel, MD,
    10. Scientific Advisory Boards:

    1. (1) SMA REACH, external advisor, 2015-19 (2) SMA Europe, scientific advisory board, 2015-19 (3) AveXis advisory board (4) Biogen advisory board (5) Roche advisory board (6) Catabasis advisory board (7) Summit advisory board (8) Capricor advisory board (9) Neurogene 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 (8) Summit: funding for travel to speak with regulatory authorities, 2015-17 (9) World Muscle Society: honorarium, 2016

    Editorial Boards:

    1. Editorial advisory board member without compensation: (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 Pediatric Neurology, 6th edition; Elsevier, 2017.

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. (1) Capricor: 2017-19 (2) Biogen: 2014-19 (3) Roche: 2015-19 (4) AveXis: 2015-19 (5) Novartis: 2015-17

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical procedures or imaging studies:

    1. Approximately half of my time and effort is devoted to patient care at Nemours Children's Hospital, mainly children with neuromuscular disorders. Some of these patients are enrolled in clinical trials. The remainder of my time is spent in teaching, research and administrative activities.

    Research Support, Commercial Entities:

    1. Dr. Richard Finkel: (1) BMS, investigator, 2016-17 (2) Biogen: advisor, 2014-17; investigator, 2015-17 (3) ReveraGen: investigator, 2016-19 (4) Cytokinetics: investigator, 2016-17 (5) AveXis: advisor, investigator, 2016-19 (7) Roche: advisor, investigator, 2016-19 (8) Summit, investigator, 2016-17 (8) Sarepta, investigator, 2015-17

    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

    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, 2018-19 Spouse recieves 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

  10. Michela Guglieri, MD,
    11. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Funding for travel and meeting registration to the World Muscle Society Conference, San Malo, October 2017 by Santhera

    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. Trial support from PTC (money paid to Newcastle University)

    Research Support, Government Entities:

    1. FOR DMD NIH funding H2020 Vision DMD grant

    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. Katharine Bushby, MD,
    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. 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

  12. Mar Tulinius, MD,
    13. Scientific Advisory Boards:

    1. Scientific advisory board for Biogen, Avexis, Sarepta Therapeutics and PTC Therapeutics

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Speaker honoraria from Biogen Avexis, Sarepta Therapeutics and PTC Therapeutics

    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

  13. Yoram Nevo, MD,
    14. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. PTC -advisory board

    Editorial Boards:

    1. NONE

    Patents:

    1. Glatiramer Acetate in muscular dystrophy

    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. Teva Pharma? Combined Copaxone and Laquinimode/pirfenidone as treatment for muscular dystrophy. PI ? Yoram Nevo Total amount 110000$ for 2 years

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. Nelly Horowitz foundation foundation Tel-Aviv University. Overexpression of kindlin-2 as a new treatment for muscular dystrophy. PI ? Yoram Nevo Total amount ? 15000 IS for 18 months David and Rebecca Decter foundation Tel-Aviv University. Overexpression of kindlin-2 as a new treatment for muscular dystrophy. PI ? Yoram Nevo Total amount ? 15000 IS for 18 months

    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

  14. Monique M. Ryan, MD,
    15. Scientific Advisory Boards:

    1. (1) Non-profit entity: Therapeutic Advisory Committee, Treat-NMD (2) Commercial entity: Data safety monitoring board for Eli-Lilly pharmaceuticals. Scientific advisory boards for Biogen, BioMarin, AveXis and PTC Therapeutics.

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Travel honoraria from Biogen Pharmaceuticals

    Editorial Boards:

    1. (1) Associate Editor, Journal of Pediatric Neurology 2007- 2014 2) Editorial Board, Journal of Clinical Neuroscience 2009- 3) Editorial board, Journal of Paediatrics and Child Health 2013-2018 4) Associate Editor, Frontiers in Neurology, 2018-

    Patents:

    1. NONE

    Publishing Royalties:

    1. Darras BT, Jones HR Jr, Ryan MM, De Vivo DC (editors). Neuromuscular Disorders of Infancy, Childhood and Adolescence: A Clinician's Approach. 2nd edition. San Diego: Academic Press, 2015.

    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) PTC Therapeutics (2) Biogen (3)BioMarin

    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. Richard Webster, MD,
    16. Scientific Advisory Boards:

    1. 1. Commercial - Data Safety and Monitoring Board Antisense Therapeutics

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. 1. Speaker Honoraria Commercial entity Australian Medical Forum GP Update 2. Speaker Monsoon Summit Kerala Association of Paediatric Neurology

    Editorial Boards:

    1. Associate Editor Neurology Journal of Paediatrics and Child Health 2016 ongoing

    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. Commerical Entity Reveragen VBP 15-002/004 - PI

    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

  16. Andrea L. Smith, MS,
    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. Employed for 2 years by TRiNDS who is the CRO for the clinical study that conducted the study management.

    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

  17. Lauren P. Morgenroth, MS,
    18. 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. Serve on non-profit: CINRG Publication and Outcomes Subcommittees and NSGC JEMF Advisory Committee

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. (1) ReveraGen (Sponsor) supports TRiNDS (current employer) for project management, data management, DSMB, and clinical outcomes activities.

    Research Support, Government Entities:

    1. 1. NIH/NINDS, 1R44NS095423-01, study coordination, 02/15/2016- 01/31/2018

    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

  18. Adrienne Arrieta, MS,
    19. 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) Employed by TRiNDS who is the CRO for the clinical study that conducted the study management and paid for by ReveraGen.

    Research Support, Government Entities:

    1. (1) Employed by TRiNDS who is the CRO for the clinical study that conducted the study management and received funding from NIH/NINDS, 1R44NS095423-01, 02/15/2016-01/31/2018.

    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

  19. Maya Shimony, MPH,
    20. 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) Employed by TRiNDS who is the CRO for the clinical study that conducted the study management and paid for by ReveraGen.

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. (1) George Washington University, (2) Employed by TRiNDS who is the CRO for the clinical study that conducted the study management and received funding from NIH/NINDS, 1R44NS095423- 01, 02/15/2016-01/31/2018.

    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

  20. Catherine Siener, PT,
    21. 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

  21. Mark Jaros, PhD,
    22. 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. Reveragen

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. Reveragen

    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

  22. Phil Shale, PhD,
    23. 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)Summit Analytical (CRO), Director of Biostatistics 2010-2019

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. (1)Summit Analytical (CRO)

    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

  23. John M. McCall, PhD,
    24. Scientific Advisory Boards:

    1. NIH Blueprint Advisory Board

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. vamorolone for treatment of muscular dystrophy

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. Reveragen Biopharma part time, board chair and VP chemistry, 8 years

    Consultancies:

    1. commercial: Hotspot, Quench, Quartet, Rodin, Rheostat, Psychogenics

    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

  24. Kanneboyina Nagaraju, PhD, DVM,
    25. Scientific Advisory Boards:

    1. Muscular dystrophy association The Myositis Association CureJM foundation

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. Frontiers in Immunology, section Autoimmune and Autoinflammatory Disorders. Neuromuscular diseases

    Patents:

    1. NON-HORMONAL STEROID MODULATORS OF NF-kB FOR TREATMENT OF DISEASE Publication number: 20190125763 Abstract: Provided herein is a pharmaceutical composition comprising a compound having the structural formula wherein the compound is present in an amount effective to treat or reduce the symptoms of muscular dystrophy. The therapeutically effective amount may be between 10 mg to 200 mg, or may be between 0.01 mg/kg to 10.0 mg/kg. Also provided are methods of treating or reducing the symptoms of muscular dystrophy, comprising the administration, to a patient in need thereof, of a therapeutically effective of the above compound. Type: Application Filed: December 19, 2018 Publication date: May 2, 2019 Inventors: John M. MCCALL, Eric HOFFMAN, Kanneboyina NAGARAJU Non-hormonal steroid modulators of NF-kB for treatment of disease Patent number: 10206933 Abstract: The present invention relates to compounds and methods which may be useful as treatments of neuromuscular diseases such as muscular dystrophy, and as inhibitors of NF-?B for the treatment or prevention of muscular wasting disease, including muscular dystrophy. Type: Grant Filed: April 10, 2017 Date of Patent: February 19, 2019 Assignee: ReveraGen Biopharma, Inc. Inventors: John M. McCall, Eric Hoffman, Kanneboyina Nagaraju Non-hormonal steroid modulators of NF-?B for treatment of disease Patent number: 10000525 Abstract: The present invention relates to compounds and methods which may be useful as treatments of neuromuscular diseases such as muscular dystrophy, and as inhibitors of NF-?B for the treatment or prevention of muscular wasting disease, including muscular dystrophy. Type: Grant Filed: November 12, 2015 Date of Patent: June 19, 2018 Assignee: ReveraGen BioPharma, Inc. Inventors: John M. McCall, Eric Hoffman, Kanneboyina Nagaraju NON-HORMONAL STEROID MODULATORS OF NF-kB FOR TREATMENT OF DISEASE Publication number: 20180015104 Abstract: The present invention relates to compounds and methods which may be useful as treatments of neuromuscular diseases such as muscular dystrophy, and as inhibitors of NF-?B for the treatment or prevention of muscular wasting disease, including muscular dystrophy. Type: Application Filed: April 10, 2017 Publication date: January 18, 2018 Inventors: John M. MCCALL, Eric HOFFMAN, Kanneboyina NAGARAJU Non-hormonal steroid modulators of NF-?B for treatment of disease Patent number: 9649320 Abstract: The present invention relates to compounds and methods which may be useful as treatments of neuromuscular diseases such as muscular dystrophy, and as inhibitors of NF-?B for the treatment or prevention of muscular wasting disease, including muscular dystrophy. Type: Grant Filed: August 5, 2016 Date of Patent: May 16, 2017 Assignee: ReveraGen Biopharma, Inc. Inventors: John M. McCall, Eric Hoffman, Kanneboyina Nagaraju NON-HORMONAL STEROID MODULATORS OF NF-kB FOR TREATMENT OF DISEASE Publication number: 20170027959 Abstract: The present invention relates to compounds and methods which may be useful as treatments of neuromuscular diseases such as muscular dystrophy, and as inhibitors of NF-?B for the treatment or prevention of muscular wasting disease, including muscular dystrophy. Type: Application Filed: August 5, 2016 Publication date: February 2, 2017 Inventors: John M. McCall, Eric Hoffman, Kanneboyina Nagaraju NON-HORMONAL STEROID MODULATORS OF NF-KAPPA BETA FOR TREATMENT OF DISEASE Publication number: 20160375037 Abstract: The present invention relates to compounds and methods which may be useful as treatments of diseases. Type: Application Filed: June 29, 2016 Publication date: December 29, 2016 Inventors: John M. McCall, Eric Hoffman, Kanneboyina Nagaraju, Jesse Damsker Non-hormonal steroid modulators of NF-?B for treatment of disease Patent number: 9434758 Abstract: The present invention relates to compounds and methods which may be useful as treatments of neuromuscular diseases such as muscular dystrophy, and as inhibitors of NF-?B for the treatment or prevention of muscular wasting disease, including muscular dystrophy. Type: Grant Filed: January 27, 2014 Date of Patent: September 6, 2016 Assignee: ReveraGen Biopharma, Inc. Inventors: John M. McCall, Eric Hoffman, Kanneboyina Nagaraju NON-HORMONAL STEROID MODULATORS OF NF-KAPPA B FOR TREATMENT OF DISEASE Publication number: 20160060289 Abstract: The present invention relates to compounds and methods which may be useful as treatments of neuromuscular diseases such as muscular dystrophy, and as inhibitors of NF-?B for the treatment or prevention of muscular wasting disease, including muscular dystrophy. Type: Application Filed: November 12, 2015 Publication date: March 3, 2016 Inventors: John M. McCall, Eric Hoffman, Kanneboyina Nagaraju Non-hormonal steroid modulators of NF-?B for treatment of disease Patent number: 9198921 Abstract: The present invention relates to compounds and methods which may be useful as treatments of neuromuscular diseases such as muscular dystrophy, and as inhibitors of NF-?B for the treatment or prevention of muscular wasting disease, including muscular dystrophy. Type: Grant Filed: April 5, 2011 Date of Patent: December 1, 2015 Assignee: ReveraGen BioPharma, Inc. Inventors: John M. McCall, Eric Hoffman, Kanneboyina Nagaraju NON-HORMONAL STEROID MODULATORS OF NF-kB FOR TREATMENT OF DISEASE Publication number: 20140142078 Abstract: The present invention relates to compounds and methods which may be useful as treatments of neuromuscular diseases such as muscular dystrophy, and as inhibitors of NF-?B for the treatment or prevention of muscular wasting disease, including muscular dystrophy. Type: Application Filed: January 27, 2014 Publication date: May 22, 2014 Applicant: ReveraGen Biopharma Inventors: John M. McCall, Eric Hoffman, Kanneboyina Nagaraju Non-hormonal steroid modulators of NF-kB for treatment of disease Patent number: 8673887 Abstract: The present invention relates to compounds and methods which may be useful for the treatment of inflammatory bowel disease, Crohn's disease and colitis. Type: Grant Filed: November 15, 2012 Date of Patent: March 18, 2014 Assignee: Reveragen Biopharma, Inc Inventors: John M. McCall, Eric Hoffman, Kanneboyina Nagaraju NON-HORMONAL STEROID MODULATORS OF NF-KB FOR TREATMENT OF DISEASE Publication number: 20130196962 Abstract: The present invention relates to compounds and methods which may be useful as treatments of neuromuscular diseases such as muscular dystrophy, and as inhibitors of NF-?B for the treatment or prevention of muscular wasting disease, including muscular dystrophy. Type: Application Filed: April 5, 2011 Publication date: August 1, 2013 Applicant: VALIDUS BIOPHARMA, INC Inventors: John M. McCall, Eric Hoffman, Kanneboyina Nagaraju Non-hormonal steroid modulators of NF-?B for treatment of disease Patent number: 8334279 Abstract: The present invention relates to compounds and methods which may be useful as treatments of neuromuscular diseases such as muscular dystrophy, and as inhibitors of NF-?B for the treatment or prevention of muscular wasting disease, including muscular dystrophy. Type: Grant Filed: December 15, 2011 Date of Patent: December 18, 2012 Assignee: Validus Genetics Inventors: John M. McCall, Eric Hoffman, Kanneboyina Nagaraju Non-hormonal steroid modulators of NF-?B for treatment of disease Patent number: 8207151 Abstract: The present invention relates to compounds and methods which may be useful as treatments of neuromuscular diseases such as muscular dystrophy, and as inhibitors of NF-?B for the treatment or prevention of muscular wasting disease, including muscular dystrophy. Type: Grant Filed: May 28, 2009 Date of Patent: June 26, 2012 Assignee: Validus Biopharma Inc. Inventors: John M. McCall, Eric Hoffman, Kanneboyina Nagaraju NON-HORMONAL STEROID MODULATORS OF NF-kB FOR TREATMENT OF DISEASE Publication number: 20120094970 Abstract: The present invention relates to compounds and methods which may be useful as treatments of neuromuscular diseases such as muscular dystrophy, and as inhibitors of NF-?B for the treatment or prevention of muscular wasting disease, including muscular dystrophy. Type: Application Filed: December 15, 2011 Publication date: April 19, 2012 Applicant: VALIDUS GENETICS Inventors: John M. McCall, Eric Hoffman, Kanneboyina Nagaraju NON-HORMONAL STEROID MODULATORS OF NF-kB FOR TREATMENT OF DISEASE Publication number: 20100087408 Abstract: The present invention relates to compounds and methods which may be useful as treatments of neuromuscular diseases such as muscular dystrophy, and as inhibitors of NF-?B for the treatment or prevention of muscular wasting disease, including muscular dystrophy. Type: Application Filed: May 28, 2009 Publication date: April 8, 2010 Applicant: VALIDUS GENETICS Inventors: John M. McCall, Eric Hoffman, Kanneboyina Nagaraju

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. Reveragen Biopharma-Co-Founder Agada biosciences-Co-Founder

    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

  25. John van den Anker, MD, PhD,
    26. Scientific Advisory Boards:

    1. (1) Commercial (Daiichi Sankyo, Endo Pharmaceuticals, Actelion Actelion) (2) Non-profit (NHLBI, EU)

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. (1) British Journal of Clinical Pharmacology, Executive E Editor, 2017-now (2) International Journal of Clinical Pharmacology & Therapeutics, Section Editor, 2010-now

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. (1) parttime employed by Reveragen Biopharma as their CMO

    Consultancies:

    1. (1) Commercial (UCB Pharma, NDA partners, Gruenenthal)

    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. Postdoctoral Research Training in Pediatric Clinical Ph Pharmacology and Therapeutics, NIH/NICHD, 1T32HD087969: 20 2016-2021; $ 206,132/yr, PI ? Bridging pharmacodynamics biomarkers to clinical outcomes i in pediatric inflammatory diseases, NIH/NICHD, 5 5U54HD090254: 2016-2021; $800,000/yr, PI

    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. Reveragen Biopharma, stock options

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  26. Laurie S. Conklin, MD,
    27. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. (1) Imaging biomarkers for managing, assessing, and treating inflammatory bowel disease. (2) Prussian Blue constructs for multimodal imaging and therapy?

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. (1) ReveraGen BioPharma, Director Clinical Programs, 2.5 years

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. (1)Genentech (family member) (2)Bristol Myers Squibb (family member) (3)Biomarker Strategies, Inc (family member)

    Research Support, Government Entities:

    1. (1) Department of Defense grant (family member) (2) NICHD U54 grant 1 U54 HD090254-01, PI, 2 years (3) NINDS SBIR 2R44NS095423-03, Co-I, 3 years

    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. Avital Cnaan, PhD,
    28. Scientific Advisory Boards:

    1. 1. Member, External Advisory Committee (EAC), NIH/NIDDK, Chronic Kidney Disease in Children, 2. Member, Pediatric Advisory Committee, FDA (end date 6/30/2018).

    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. TRiNDS, Board member.

    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

  28. Heather Gordish-Dressman, PhD,
    29. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. (1) TREAT-NMD Advisory Committee on Therapeutics, honoraria and travel

    Editorial Boards:

    1. (1) Journal of Intensive Care Medicine, Editorial board member, 2014 to present

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. (1) AGADA BioSciences (2) SOLID-GT

    Speakers' Bureaus:

    1. NONE

    Other activities:

    1. (1) TRiNDS, LLC, co-founder and Board of Directors president

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. (1) Santhera Pharmaceuticals

    Research Support, Government Entities:

    1. (1) NICHD, 5U54HD090257-03, statistician, 2016-present (2) NIH, 5U54HD090254-03, statistician, 2016-present (3) DoD (Binghamton Univ.), W81XWH16-1-0557, statistician, 2017-present (4) DoD (Binghamton Univ.), W81XWH-16-1-0572, statistician, 2017-present (5)

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. (1) TRiNDS, LLC.

    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. Jesse M. Damsker, PhD,
    30. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. Inventor on patents related to vamorolone methods of use claims

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. I am an employee of ReveraGen BioPharma 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. Small business grants from the National Institutes of Health

    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

  30. Paula R. Clemens, MD;
    31. Scientific Advisory Boards:

    1. (1) Pfizer DSMB; (2) NIH DSMB; Advisory boards for (3) Sanofi, (4) Roche, (5) Spark, (6) UCB Biopharma

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Travel funding by (1) Muscular Dystrophy Association, (2) Amicus Therapeutics, (3) Sanofi

    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) NS Pharma, (2) ReveraGen, (3) Sanofi, (4) Amicus

    Research Support, Government Entities:

    1. (1) NIH 1R01AR071369-01,co-I, 2017-2022, (2) NIH 2R44NS095423-03, co-PI, 2018-2020, (3) NIH 1U24NS107166- 01, PD, 2018-2023, (4) NIH 1R44NS095423-01, co-PI, 2016- 2018

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. Muscular Dystrophy Association

    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. the Cooperative International Neuromuscular Research Group
  1. From ReveraGen Biopharma (E.P.H., J.M.M., K.N., J.v.d.A., L.S.C., J.M.D.), Rockville, MD; Binghamton University–SUNY (E.P.H., K.N.), NY: Camden Group (B.D.S., L.J.M.-G.), LLC, St. Louis, MO; Duke University (E.C.S.), Durham, NC; University of Texas Southwestern (D.C.), Dallas; Alberta Children's Hospital (J.K.M.), Calgary, Canada; University of California Davis (C.M.M.), Sacramento; Ann & Robert H. Lurie Children's Hospital (N.L.K.), Chicago, IL; Nemours Children's Hospital (R.S.F.), Orlando, FL; John Walton Muscular Dystrophy Research Centre (M.G., K.B.), Newcastle University, Newcastle-Upon-Tyne, UK; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Royal Children's Hospital and Murdoch Children's Research Institute (M.M.R.), Melbourne, Australia; The Children's Hospital at Westmead (R.W.), Sydney, Australia; TRiNDS LLC (A.L.S., L.P.M., A.A., M.S., C.S.), Kensington, MD; Summit Analytical (M.J., P.S.), Denver, CO; Children's National Health System (J.v.d.A., L.S.C., A.C., H.G.-D.), Washington, DC; and University of Pittsburgh and Department of Veterans Affairs Medical Center (P.R.C.), PA.
  1. Correspondence
    Dr. Hoffman ericphoffman{at}gmail.com
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