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April 14, 2020; 94 (15 Supplement) Tuesday, April 28

FIREFISH Part 2: Efficacy and Safety of Risdiplam (RG7916) in Infants with Type 1 Spinal Muscular Atrophy (SMA) (1302)

Laurent Servais, Giovanni Baranello, Riccardo Masson, Maria Mazurkiewicz-Bełdzińska, Kristy Rose, Dmitry Vlodavets, Hui Xiong, Edmar Zanoteli, Muna El-Khairi, Sabine Fuerst-Recktenwald, Marianne Gerber, Ksenija Gorni, Heidemarie Kletzl, Renata Scalco, Basil T. Darras
First published April 14, 2020,
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Article Information

vol. 94 no. 15 Supplement 1302
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. Laurent Servais1,2,
    2. Giovanni Baranello3,4,
    3. Riccardo Masson3,
    4. Maria Mazurkiewicz-Bełdzińska5,
    5. Kristy Rose6,
    6. Dmitry Vlodavets7,
    7. Hui Xiong8,
    8. Edmar Zanoteli9,
    9. Muna El-Khairi10,
    10. Sabine Fuerst-Recktenwald11,
    11. Marianne Gerber12,
    12. Ksenija Gorni13,
    13. Heidemarie Kletzl12,
    14. Renata Scalco11 and
    15. Basil T. Darras
    16. on behalf of the FIREFISH Working Group14
  1. Laurent Servais1,2,
  2. Giovanni Baranello3,4,
  3. Riccardo Masson3,
  4. Maria Mazurkiewicz-Bełdzińska5,
  5. Kristy Rose6,
  6. Dmitry Vlodavets7,
  7. Hui Xiong8,
  8. Edmar Zanoteli9,
  9. Muna El-Khairi10,
  10. Sabine Fuerst-Recktenwald11,
  11. Marianne Gerber12,
  12. Ksenija Gorni13,
  13. Heidemarie Kletzl12,
  14. Renata Scalco11 and
  15. Basil T. Darras
  1. 1Reference Center for Neuromuscular Disease, Centre Hospitalier Régional de La Citadelle
  2. 2MDUK Oxford Neuromuscular Centre
  3. 3Developmental Neurology Unit, Carlo Besta Neurological Research Institute Foundation
  4. 4The Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health
  5. 5Department of Developmental Neurology, Medical University of Gdansk
  6. 6Paediatric Gait Analysis Service of New South Wales, The Children’s Hospital at Westmead
  7. 7Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Insitute of Pirogov Russian National Research Medical University
  8. 8Department of Pediatrics, Peking University First Hospital
  9. 9Hospital das Clinicas
  10. 10Roche Products Ltd.
  11. 11Pharma Development Neurology, F. Hoffmann-La Roche Ltd.
  12. 12Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel
  13. 13PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd.
  14. 14Boston Children’s Hospital, Harvard Medical School

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