Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial
Thomas O. Crawford(Johns Hopkins University), Barbara Steinborn, Craig M. Zaidman(Washington University in St. Louis), Kaitlin Batley(The University of Texas Southwestern Medical Center), W. Ludo van der Pol(Utrecht University), A. Seferian(Sorbonne Université), Nancy L. Kuntz(Lurie Children's Hospital), Valeria Sansone(University of Milan), Carolina Tesi Rocha(Stanford University), Saunder Bernes, C. Cancès(Université Fédérale de Toulouse Midi-Pyrénées), Sabrina W. Yum(Children's Hospital of Philadelphia), Sally Dunaway Young(Palo Alto University), Guochen Song(Scholar Rock (United States)), Sonia Messina(University of Messina), Liesbeth De Waele(KU Leuven), Eugenio Mercuri(Università Cattolica del Sacro Cuore), Giridhar Tirucherai(Bristol-Myers Squibb (United States)), Astrid Blaschek(Ludwig-Maximilians-Universität München), Astrid Pechmann(University of Freiburg), Katherine D. Mathews(University of Iowa), Jessica Nance(Johns Hopkins University), Guolin Zhao(Scholar Rock (United States)), Jing L. Marantz(Scholar Rock (United States)), Crystal M. Proud(Children's Hospital of The King's Daughters), Russell J. Butterfield(University of Utah), Jeffrey Statland(The University of Texas at San Antonio Health Science Center), Michael S. Cartwright(Atrium Health Wake Forest Baptist), Seth J. Perlman(Seattle Children's Hospital), Jennifer M. Kwon(University of Wisconsin–Madison), Jena M. Krueger(Helen DeVos Children's Hospital), R. H. Fryer, Leigh Ramos‐Platt, Basil T. Darras, Heike Kölbel(University of Duisburg-Essen), Elena Mazzone(Università Cattolica del Sacro Cuore), Richard S. Finkel(St. Jude Children's Research Hospital), Dorothea Holzwarth, Anne M. Connolly(Washington University in St. Louis), Randal Richardson(Gillette Children's Specialty Healthcare), José Rosselló(Scholar Rock (United States)), Julie Parsons, Marta Gómez García de la Banda(Vall d'Hebron Hospital Universitari), H. Phan(Rare Disease Therapeutics (United States)), Claudio Bruno(Istituto Giannina Gaslini), Riccardo Masson, Chamindra Laverty Rady, Bert Yao(Scholar Rock (United States))
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