Neuropediatrics 2019; 50(S 02): S1-S55
DOI: 10.1055/s-0039-1698170
Oral Presentations
Neuromuscular Diseases and Varia
Georg Thieme Verlag KG Stuttgart · New York

Onasemnogene Abeparvovec Gene-Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 1 (SMA1): Pivotal Phase 3 Study (STR1VE) Update

Authors

  • John W. Day

    1   Stanford University Medical Center, Department of Neurology, Stanford, Californai, United States

  • Claudia A. Chiriboga

    2   Columbia University Medical Center, Division of Pediatric Neurology, New York, New York, United States

  • Thomas O. Crawford

    3   Johns Hopkins Medicine, Department of Neurology, Baltimore, Maryland, United States

  • Basil T. Darras

    4   Boston Children’s Hospital, Department of Neurology, Boston, Massachusetts, United States

  • Richard S. Finkel

    5   Nemours Children’s Hospital, Division of Neurology Department of Pediatrics, Orlando, Florida, United States

  • Anne M. Connolly

    6   Washington University School of Medicine, Department of Neurology, St. Louis, Missouri, United States

  • Susan T. Iannaccone

    7   University of Texas Southwestern Medical Center, Department of Pediatrics, Dallas, Texas, United States

  • Nancy L. Kuntz

    8   Ann and Robert H. Lurie Children’s Hospital of Chicago, Division of Neurology, Chicago, Illinois, United States

  • Loren Peña

    9   University of Cincinnati Children’s Hospital, Division of Human Genetics, Cincinnati, Ohio, United States

  • Meredith Schultz

    10   AveXis, Inc., AveXis, Inc., Bannockburn, Illinois, United States

  • Perry B. Shieh

    11   David Geffen School of Medicine at UCLA, Department of Neurology, Los Angeles, California, United States

  • Edward C. Smith

    12   Duke University School of Medicine, Department of Pediatrics, Durham, North Carolina, United States

  • Uwe Ernst

    13   AveXis, Inc., Medical Affairs, Bannockburn, Illinois, United States

  • Douglas E. Feltner

    14   AveXis, Inc., Clinical Development, Bannockburn, Illinois, United States

  • Francis G. Orgrinc

    15   AveXis, Inc., Biostatistics, Biostatistics, United States

  • Ankita Shah

    16   AveXis, Inc., Clinical Operations, Bannockburn, Illinois, United States

  • Haojun Ouyang

    17   AveXis, Inc., Biostatistics, Clinical Development, Bannockburn, Illinois, United States

  • Thomas A. Macek

    14   AveXis, Inc., Clinical Development, Bannockburn, Illinois, United States

  • Elaine Kernbauer

    16   AveXis, Inc., Clinical Operations, Bannockburn, Illinois, United States

  • James L’Italien

    18   AveXis, Inc., Regulatory and Quality, Bannockburn, Illinois, United States

  • Douglas M. Sproule

    14   AveXis, Inc., Clinical Development, Bannockburn, Illinois, United States

  • Brian K. Kaspar

    19   AveXis, Inc., Pre-clinical, Bannockburn, Illinois, United States

  • Jerry R. Medel

    20   Nationwide Children’s Hospital, Center for Gene Therapy, Columbus, Ohio, United States
Weitere Informationen

Publikationsverlauf

Publikationsdatum:
11. September 2019 (online)

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Research Question: SMA1 is a rapidly progressing neurologic disease caused by biallelic survival motor neuron 1 gene (SMN1) deletion/mutation. The one-time SMN GRT onasemnogene abeparvovec (AVXS-101) addresses the genetic root cause of SMA. In a phase 1/2a study (NCT02122952), AVXS-101 resulted in exceptional event-free survival, motor function improvements, and motor milestone achievement in patients with SMA1. We report data from the multicenter, open-label, pivotal phase 3 STR1VE study (NCT03306277) investigating intravenous AVXS-101 in SMA1 patients (bi-allelic SMN1 mutations/deletions, 2xSMN2) aged