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European Journal of Haematology|September 15, 2023
Long-term effectiveness of eculizumab: Data from the International PNH RegistryLouis Terriou, Jong Wook Lee, Cecily Forsyth, et al.
Blood Cells, Molecules & Diseases|May 20, 2014
Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4 years of treatmentElena Lukina, Nora Watman, Marta Dragosky, et al.
European Journal of Haematology|May 3, 2018
Prognostic markers in core-binding factor AML and improved survival with multiple consolidation cycles of intermediate-/high-dose cytarabineAshvind Prabahran, Mark Tacey, Shaun Fleming, et al.
Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation|July 21, 2007
Hematopoietic stem cell transplantation in Australia and New Zealand, 1992-2004Ian Nivison-Smith, Kenneth F Bradstock, Anthony J Dodds, et al.
Orphanet Journal of Rare Diseases|April 22, 2015
Effect of cyclic, low dose pyrimethamine treatment in patients with Late Onset Tay Sachs: an open label, extended pilot studyEtty Osher, Aviva Fattal-Valevski, Liora Sagie, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|March 7, 2009
Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1Gregory A Grabowski, Katherine Kacena, J Alexander Cole, et al.
American Journal of Hematology|April 2, 2024
A 10-year follow-up of high-dose ambroxol treatment combined with enzyme replacement therapy for neuropathic Gaucher diseaseSoojin Hwang, Hyunwoo Bae, Ji-Hee Yoon, et al.
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