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 FinkelClaudia 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 Mendell Tip Tip