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April 14, 2020; 94 (15 Supplement) Sunday, April 26

ASPIRO Gene Therapy Trial In X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Safety And Efficacy Findings up to 72 Weeks Post-Treatment (1053)

Perry B. Shieh, Nancy Kuntz, Barbara Smith, James Dowling, Wolfgang Müller-Felber, Carsten G. Bönnemann, Laurent Servais, Francesco Muntoni, Astrid Blaschek, Sarah Neuhaus, Lindsay N. Alfano, Alan H. Beggs, Ana Buj-Bello, Martin (Casey) Childers, Tina Duong, Robert J. Graham, Minal Jain, Emma S. James, Michael W. Lawlor, Jun Lee, Victoria MacBean, Fulvio Mavilio, Michael Murtagh, Mojtaba Noursalehi, Suyash Prasad, Salvador Rico
First published April 14, 2020,
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Article Information

vol. 94 no. 15 Supplement 1053
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 April 14, 2020.
Copyright & Usage: 
© 2020

Author Disclosures

    1. Perry B. Shieh1,
    2. Nancy Kuntz2,
    3. Barbara Smith3,
    4. James Dowling4,
    5. Wolfgang Müller-Felber5,
    6. Carsten G. Bönnemann6,
    7. Laurent Servais7,
    8. Francesco Muntoni8,
    9. Astrid Blaschek5,
    10. Sarah Neuhaus6,
    11. Lindsay N. Alfano9,
    12. Alan H. Beggs10,
    13. Ana Buj-Bello11,
    14. Martin (Casey) Childers12,
    15. Tina Duong13,
    16. Robert J. Graham10,
    17. Minal Jain14,
    18. Emma S. James15,
    19. Michael W. Lawlor16,
    20. Jun Lee15,
    21. Victoria MacBean17,
    22. Fulvio Mavilio15,
    23. Michael Murtagh15,
    24. Mojtaba Noursalehi15,
    25. Suyash Prasad18 and
    26. Salvador Rico15
  1. Perry B. Shieh1,
  2. Nancy Kuntz2,
  3. Barbara Smith3,
  4. James Dowling4,
  5. Wolfgang Müller-Felber5,
  6. Carsten G. Bönnemann6,
  7. Laurent Servais7,
  8. Francesco Muntoni8,
  9. Astrid Blaschek5,
  10. Sarah Neuhaus6,
  11. Lindsay N. Alfano9,
  12. Alan H. Beggs10,
  13. Ana Buj-Bello11,
  14. Martin (Casey) Childers12,
  15. Tina Duong13,
  16. Robert J. Graham10,
  17. Minal Jain14,
  18. Emma S. James15,
  19. Michael W. Lawlor16,
  20. Jun Lee15,
  21. Victoria MacBean17,
  22. Fulvio Mavilio15,
  23. Michael Murtagh15,
  24. Mojtaba Noursalehi15,
  25. Suyash Prasad18 and
  26. Salvador Rico15
  1. 1University of California
  2. 2Ann & Robert H Lurie Children's Hospital of Chicago
  3. 3University of Florida
  4. 4Hospital for Sick Children
  5. 5Klinikum der Universität München
  6. 6NIH Porter Neuroscience Research Center
  7. 7Hôpital Armand Trousseau
  8. 8Great Ormond Street Hospital for Children
  9. 9Nationwide Children’s Hospital
  10. 10Children’s Hospital Boston
  11. 11Généthon
  12. 12AskBio
  13. 13Stanford University
  14. 14NIH Hatfield Clinical Research Center
  15. 15Audentes Therapeutics
  16. 16Medical College of Wisconsin
  17. 17Brunel University London
  18. 18Formerly Audentes Therapeutics

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