Long-term hematological, visceral, and growth outcomes in children with Gaucher disease type 3 treated with imiglucerase in the International Collaborative Gaucher Group Gaucher Registry
El-Beshlawy A, Tylki-Szymanska A, Vellodi A, Belmatoug N, Grabowski GA, Kolodny EH, Batista JL, Cox GF, Mistry PK. Long-term hematological, visceral, and growth outcomes in children with Gaucher disease type 3 treated with imiglucerase in the International Collaborative Gaucher Group Gaucher Registry. Molecular Genetics And Metabolism 2016, 120: 47-56. PMID: 28040394, DOI: 10.1016/j.ymgme.2016.12.001.Peer-Reviewed Original ResearchConceptsEnzyme replacement therapyInternational Collaborative Gaucher Group Gaucher RegistryGD3 patientsGaucher RegistryPrimary central nervous system involvementImiglucerase enzyme replacement therapyCentral nervous system involvementGaucher diseaseSingle-center seriesGrowth outcomesNervous system involvementGaucher disease type 3Height z-scoreNumber of patientsLife-prolonging benefitsBroad phenotypic spectrumImiglucerase treatmentVisceral diseaseHemoglobin levelsPlatelet countReplacement therapySevere anemiaVisceral manifestationsSpleen volumeSystem involvement