Transformation in Pre-Treatment Presentations of Gaucher Disease during the First Two Decades of Imiglucerase Enzyme Replacement Therapy: A Report from the International Collaborative Gaucher Group Gaucher Registry
Mistry P, Weinreb N, Batista J, Andersson H, Balwani M, Burrow T, Charrow J, Kaplan P, Khan A, Kishnani P, Kolodny E, Rosenbloom B, Scott C. Transformation in Pre-Treatment Presentations of Gaucher Disease during the First Two Decades of Imiglucerase Enzyme Replacement Therapy: A Report from the International Collaborative Gaucher Group Gaucher Registry. Blood 2016, 128: 4877. DOI: 10.1182/blood.v128.22.4877.4877.Peer-Reviewed Original ResearchImiglucerase enzyme replacement therapyGaucher disease type 1ICGG Gaucher RegistryEnzyme replacement therapyInternational Collaborative Gaucher Group Gaucher RegistrySanofi GenzymeERT initiationGaucher RegistryBone crisesPfizer IncAge groupsSpeakers bureauBone painSplenectomy statusReplacement therapyPrevalent symptomsIntact spleenHematologic parametersExact testAdvisory CommitteeNon-splenectomized groupNon-splenectomized patientsFisher's exact testOutcomes Research InstituteDisease type 1