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  5. van Beers, Eduard J. [Author]; Straaten, Stephanie van [Author]; Morton, D. Holmes [Author]; Barcellini, Wilma [Author]; Eber, Stefan W. [Author]; Glader, Bertil [Author]; Yaish, Hassan M. [Author]; Chonat, Satheesh [Author]; Kwiatkowski, Janet L. [Author]; Rothman, Jennifer A. [Author]; Sharma, Mukta [Author]; Neufeld, Ellis J. [Author]; Sheth, Sujit [Author]; Despotovic, Jenny M. [Author]; Kollmar, Nina [Author]; Pospíšilová, Dagmar [Author]; Knoll, Christine M. [Author]; Kuo, Kevin [Author]; Pastore, Yves D. [Author]; Thompson, Alexis A. [Author]; Newburger, Peter E. [Author]; Ravindranath, Yaddanapudi [Author]; Wang, Winfred C. [Author]; Wlodarski, Marcin W. [Author]; [...]

    Prevalence and management of iron overload in pyruvate kinase deficiency: report from the Pyruvate kinase deficiency natural history study

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    Pavia: Ferrata Storti Foundation, 2019

    Published in: Haematologica ; 104, 2 (2019), e51-e53$z1592-8721

  10. Grace, Rachael F.; Xue, Xiaoqiang; Jamieson, Brian

    Rationale and Design of a Phase 3b Multicenter, Randomized, Double-Blind Placebo-Controlled, Parallel-Group Trial with an Open-Label Extension Phase to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Pediatric Patients with Immune Thrombocytopenia

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    American Society of Hematology, 2021

    Published in: Blood, 138 (2021) Supplement 1, Seite 4211-4211