2008
Management of non‐neuronopathic Gaucher disease with special reference to pregnancy, splenectomy, bisphosphonate therapy, use of biomarkers and bone disease monitoring
Cox TM, Aerts JM, Belmatoug N, Cappellini MD, vom Dahl S, Goldblatt J, Grabowski GA, Hollak CE, Hwu P, Maas M, Martins AM, Mistry PK, Pastores GM, Tylki‐Szymanska A, Yee J, Weinreb N. Management of non‐neuronopathic Gaucher disease with special reference to pregnancy, splenectomy, bisphosphonate therapy, use of biomarkers and bone disease monitoring. Journal Of Inherited Metabolic Disease 2008, 31: 319-336. PMID: 18509745, DOI: 10.1007/s10545-008-0779-z.Peer-Reviewed Original ResearchMeSH KeywordsAbsorptiometry, PhotonBiomarkersBone DiseasesDiphosphonatesFemaleGaucher DiseaseHumansMagnetic Resonance ImagingPregnancyPregnancy ComplicationsSplenectomyConceptsNon-neuronopathic Gaucher diseaseLong-term disease outcomesGaucher diseaseBone marrow infiltrationManagement of pregnancyAchievable treatment goalsUse of biomarkersBisphosphonate therapyBisphosphonate treatmentMarrow infiltrationTherapeutic eraDisease management approachDisease outcomeTreatment goalsBone diseaseContemporary clinical researchEnzyme replacementBiochemical markersDisease severityClinical researchDiseaseDisease monitoringSplenectomyAppropriate usePregnancy
2004
Enzyme replacement therapy and monitoring for children with type 1 Gaucher disease: consensus recommendations
Charrow J, Andersson HC, Kaplan P, Kolodny EH, Mistry P, Pastores G, Prakash-Cheng A, Rosenbloom BE, Scott CR, Wappner RS, Weinreb NJ. Enzyme replacement therapy and monitoring for children with type 1 Gaucher disease: consensus recommendations. The Journal Of Pediatrics 2004, 144: 112-120. PMID: 14722528, DOI: 10.1016/j.jpeds.2003.10.067.Peer-Reviewed Original Research