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Onasemnogene abeparvovec gene therapy for symptomatic inf... | ResearchHub
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Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial
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Authors
John Day
•
Richard Finkel
18 more
•
Jerry Mendell
Published
March 18, 2021
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Journal
The Lancet Neurology
Topics
Internal Medicine
Pediatrics
Surgery
Medicine
Internal Medicine
Pediatrics
Surgery
Genetics
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DOI
10.1016/s1474-4422(21)00001-6
