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Genotype/phenotype correlations of childhood-onset congenital sideroblastic anaemia in a European cohort Fouquet, Cyrielle; Le Rouzic, Marie-Amelyne; Leblanc, Thierry; Fouyssac, Fanny; ... Ducassou, Stephane 2019
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Intra-monocyte Pharmacokinetics of Imiglucerase Supports a Possible Personalized Management of Gaucher Disease Type 1 Berger, Juliette; Vigan, Marie; Pereira, Bruno; Nguyen, Thu Thuy; ... Berger, Marc G 2019
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Impact of imiglucerase supply constraint on the therapeutic management and course of disease in French patients with Gaucher disease type 1 Stirnemann, Jérôme; Rose, Christian; De Roux Serratrice, Christine; Dalbies, Florence; ... Belmatoug, Nadia 2015
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The French Gaucher's disease registry: clinical characteristics, complications and treatment of 562 patients Stirnemann, Jérôme; Vigan, Marie; Hamroun, Dalil; Heraoui, Djazia; ... Belmatoug, Nadia 2012