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
John W Day(Stanford Medicine), Richard S. Finkel(St. Jude Children's Research Hospital), Claudia A. Chiriboga(Columbia University Irving Medical Center), Anne M. Connolly(Nationwide Children's Hospital), Thomas O. Crawford(Johns Hopkins University), Basil T. Darras(Boston Children's Hospital), Susan T. Iannaccone(The University of Texas Southwestern Medical Center), Nancy L. Kuntz(Lurie Children's Hospital), Loren D.M. Peña(Cincinnati Children's Hospital Medical Center), Perry B. Shieh(University of California, Los Angeles), Edward C. Smith(Duke University), Jennifer M. Kwon(University of Wisconsin–Madison), Craig M. Zaidman(Washington University in St. Louis), Meredith Schultz(Novartis (United States)), Douglas E. Feltner(Novartis (United States)), Sitra Tauscher‐Wisniewski(Novartis (United States)), Haojun Ouyang(Novartis (United States)), Deepa H. Chand(Washington University in St. Louis), Douglas M. Sproule(Novartis (United States)), Thomas A. Macek(Novartis (United States)), Jerry R. Mendell(Nationwide Children's Hospital)
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