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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
Authors
John Day,Richard Finkel
Claudia Chiriboga,Anne Connolly,Thomas Crawford,Basil Darras,Susan Iannaccone,Nancy Kuntz,Loren Peña,Perry Shieh,Edward Smith,Jennifer Kwon,Craig Zaidman,Meredith Schultz,Douglas Feltner,Sitra Tauscher‐Wisniewski,Haojun Ouyang,Deepa Chand,Douglas Sproule,Thomas Macek
+18 authors
,Jerry MendellJournal
Published
Mar 18, 2021
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