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Transplantation|August 8, 2014
Validating the allogeneic stem cell transplantation disease risk index: sample size, follow-up, and local data are importantAndrew B M Lim, Andrew W Roberts, Kate Mason, et al.
Bone Marrow Transplantation|October 5, 2018
Current use of biosimilar G-CSF for haematopoietic stem cell mobilisationSimon Pahnke, Torstein Egeland, Jörg Halter, et al.
International Journal of Molecular Sciences|July 24, 2021
Impact of Long-Term Enzyme Replacement Therapy on Glucosylsphingosine (Lyso-Gb1) Values in Patients with Type 1 Gaucher Disease: Statistical Models for Comparing Three Enzymatic FormulationsTama Dinur, Ulrike Grittner, Shoshana Revel-Vilk, et al.
Internal Medicine Journal|June 14, 2017
Clinical benefit of eculizumab in patients with no transfusion history in the International Paroxysmal Nocturnal Haemoglobinuria RegistryAntonio M Almeida, Camille Bedrosian, Alexander Cole, et al.
Molecular Genetics and Metabolism|August 31, 2017
Reductions in glucosylsphingosine (lyso-Gb1) in treatment-naïve and previously treated patients receiving velaglucerase alfa for type 1 Gaucher disease: Data from phase 3 clinical trialsDeborah Elstein, Björn Mellgard, Quinn Dinh, et al.
Calcified Tissue International|November 8, 2014
Gaucher disease and bone manifestationsGemma Marcucci, Ari Zimran, Bruno Bembi, et al.
JAMA|September 20, 2007
Carrier screening for Gaucher disease: lessons for low-penetrance, treatable diseasesShachar Zuckerman, Amnon Lahad, Amir Shmueli, et al.
American Journal of Kidney Diseases : the Official Journal of the National Kidney Foundation|October 29, 2005
A comprehensive assessment of renal function in patients with Gaucher diseaseRachel Becker-Cohen, Deborah Elstein, Ayala Abrahamov, et al.
Molecular Genetics and Metabolism Reports|January 13, 2018
Characteristics of 26 patients with type 3 Gaucher disease: A descriptive analysis from the Gaucher Outcome SurveyIda Vanessa D Schwartz, Özlem Göker-Alpan, Priya S Kishnani, et al.
Rheumatology International|March 5, 2008
Molecular aspects of osteopathy in type 1 Gaucher disease: correlation between genetics and bone densityEfrat Arnheim, Gaya Chicco, Mici Phillips, et al.
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