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Journal of Clinical Medicine|May 25, 2024
Long-Term Treatment of Gaucher Disease with Velaglucerase Alfa in ERT-Naïve Patients from the Gaucher Outcome Survey (GOS) RegistryPatrick Deegan, Heather Lau, Deborah Elstein, et al.Journal of Clinical Medicine|June 28, 2023
Reply to Mistry et al. The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on "Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158"Derralynn A Hughes, Patrick Deegan, Pilar Giraldo, et al.Journal of Clinical Medicine|September 9, 2022
Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS)Derralynn A Hughes, Patrick Deegan, Pilar Giraldo, et al.Blood Cells, Molecules & Diseases|May 21, 2013
Miglustat therapy in type 1 Gaucher disease: clinical and safety outcomes in a multicenter retrospective cohort studyDavid J Kuter, Atul Mehta, Carla E M Hollak, et al.Human Mutation|August 6, 2013
Characterization of variants in the glucosylceramide synthase gene and their association with type 1 Gaucher disease severityPilar Alfonso, Joaquín Navascués, Silvia Navarro, et al.Pediatric Research|June 27, 2002
Insulin-like growth factors in childhood-onset Gaucher diseaseSegundo Rite, Antonio Baldellou, Pilar Giraldo, et al.Archives of Medical Research|June 17, 2021
Gaucher Disease: Identification and Novel Variants in Mexican and Spanish PatientsRaúl Silva García, Laura López de Frutos, Elsa Ávila Arreguin, et al.Brain : a Journal of Neurology|August 10, 2010
Peripheral neuropathy in adult type 1 Gaucher disease: a 2-year prospective observational studyMarieke Biegstraaten, Eugen Mengel, Laszlo Maródi, et al.Haematologica|April 19, 2012
Myelodysplasia-associated immunophenotypic alterations of bone marrow cells in myeloma: are they present at diagnosis or are they induced by lenalidomide?Sergio Matarraz, Bruno Paiva, María Diez-Campelo, et al.Blood Cells, Molecules & Diseases|November 26, 2015
Patients with type 1 Gaucher disease in Spain: A cross-sectional evaluation of health statusPilar Giraldo, Jordi Pérez-López, Ramiro Núñez, et al.Pageof 16