Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study

Darryl C. De Vivo(Columbia University Irving Medical Center), Wildon Farwell(Biogen (United States)), Douglas A. Kerr(Tampa General Hospital), Gabriel Braley(Biogen (United States)), Janbernd Kirschner(University of Freiburg), Monique M. Ryan(Royal Children's Hospital), Nancy L. Kuntz(Lurie Children's Hospital), Valeria Sansone(University of Milan), Thomas O. Crawford(Johns Hopkins University), Stephanie Fradette(Biogen (United States)), Francy Shu(University of California, Los Angeles), Yuh‐Jyh Jong(National Yang Ming Chiao Tung University), Marco Petrillo(Biogen (United States)), Wuh‐Liang Hwu(MRC Epidemiology Unit), Tawfeg Ben‐Omran(Qatar Airways (Qatar)), Christopher Stebbins(University of Illinois Chicago), Enrico Bertini(Bambino Gesù Children's Hospital), Sandra P. Reyna(Novartis (United States)), Russell J. Butterfield(University of Utah), Ishir Bhan(Harvard University Press), Sarah Gheuens(Biogen (United States)), Kathryn J. Swoboda(Massachusetts General Hospital), John F. Staropoli(Biogen (United States)), Richard S. Finkel(St. Jude Children's Research Hospital), Haluk Topaloğlu(Yeditepe University), Julie Parsons, Alfred Sandrock(Biogen (United States)), Kristina Johnson(Biogen (United States)), Richard Foster(Massachusetts Institute of Technology)
Neuromuscular Disorders
September 12, 2019
Cited by 636


Related Papers