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Double-blind, randomized, multicenter phase 2 study of SC411 in children with sickle cell disease (SCOT trial) by Ahmed A. Daak, Carlton D. Dampier, Beng Fuh, Julie Kanter, Ofelia A. Alvarez, L. Vandy Black, Melissa A. McNaull, Michael U. Callaghan, Alex George, Lynne Neumayr, Lee M. Hilliard, Fredrick Sancilio, Adrian L. Rabinowicz, Matthew M. Heeney
Published 2018-08-01
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The phenotypic spectrum of germline YARS2 variants: from isolated sideroblastic anemia to mitochondrial myopathy, lactic acidosis and sideroblastic anemia 2 by Lisa G. Riley, Matthew M. Heeney, Joëlle Rudinger-Thirion, Magali Frugier, Dean R. Campagna, Ronghao Zhou, Gregory A. Hale, Lee M. Hilliard, Joel A. Kaplan, Janet L. Kwiatkowski, Colin A. Sieff, David P. Steensma, Alexander J. Rennings, Annet Simons, Nicolaas Schaap, Richard J. Roodenburg, Tjitske Kleefstra, Leonor Arenillas, Josep Fita-Torró, Rasha Ahmed, Miguel Abboud, Elie Bechara, Roula Farah, Rienk Y. J. Tamminga, Sylvia S. Bottomley, Mayka Sanchez, Gerwin Huls, Dorine W. Swinkels, John Christodoulou, Mark D. Fleming
Published 2018-12-01
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