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Case Report: Eltrombopag in mosaic and gene therapy-treated patients with Fanconi anemia by Josune Zubicaray, June Iriondo, Elena Sebastián, Alejandro Sanz, Paula Rio, Jean Soulier, Sonsoles San Román, José J. Uriz, Susana Navarro, Eileen Nicoletti, Juan A. Bueren, Jonathan D. Schwartz, Julián Sevilla
Published 2025-08-01Get full text
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Disease characteristics and outcomes of acute myeloid leukemia in germline RUNX1 deficiency (Familial Platelet Disorder with associated Myeloid Malignancy) by Martijn P. T. Ernst, Jurjen Versluis, Peter J. M. Valk, Marc Bierings, Rienk Y. J. Tamminga, Louise H. Hooimeijer, Konstanze Döhner, Paolo Gresele, Kiran Tawana, Saskia M. C. Langemeijer, Bert A. Van der Reijden, Helena Podgornik, Matjaz Sever, Tor H. A. Tvedt, Tom Vulliamy, Jude Fitzgibbon, Inderjeet Dokal, Panagiotis Baliakas, José M. Bastida, Christian Pohlkamp, Torsten Haferlach, Lise Larcher, Jean Soulier, Roger E. G. Schutgens, Kathleen Freson, Nicolas Duployez, Bob Löwenberg, Katrin Ericson, Jörg Cammenga, Tim Ripperger, Marc H. G. P. Raaijmakers
Published 2025-01-01Get full text
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