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

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
› Author Affiliations
Further Information

Publication History

Publication Date:
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


     

    No conflict of interest has been declared by the author(s).