Enzyme replacement therapy and monitoring for children with type 1 Gaucher disease: consensus recommendations
Open Access
- 31 January 2004
- journal article
- review article
- Published by Elsevier in The Journal of Pediatrics
- Vol. 144 (1), 112-120
- https://doi.org/10.1016/j.jpeds.2003.10.067
Abstract
No abstract availableKeywords
This publication has 43 references indexed in Scilit:
- Analysis and Classification of 304 Mutant Alleles in Patients with Type 1 and Type 3 Gaucher DiseaseAmerican Journal of Human Genetics, 2000
- Acceleration of retarded growth in children with Gaucher disease after treatment with algluceraseThe Journal of Pediatrics, 1996
- Sequence and rate of bone marrow conversion in the femora of children as seen on MR imaging: are accepted standards accurate?American Journal of Roentgenology, 1994
- Marked elevation of plasma chitotriosidase activity. A novel hallmark of Gaucher disease.Journal of Clinical Investigation, 1994
- Dose-dependent responses to macrophage-targeted glucocerebrosidase in a child with Gaucher diseaseThe Journal of Pediatrics, 1992
- Hematologic improvement in a patient with gaucher disease on long‐term enzyme replacement therapy: Evidence for decreased splenic sequestration and improved red blood cell survivalAmerican Journal of Hematology, 1991
- Clinical phenotype of Gaucher disease in relation to properties of mutant glucocerebrosidase in cultured fibroblastsBiochimica et Biophysica Acta (BBA) - Molecular Basis of Disease, 1991
- Replacement Therapy for Inherited Enzyme Deficiency — Macrophage-Targeted Glucocerebrosidase for Gaucher's DiseaseNew England Journal of Medicine, 1991
- Mutation analysis of an Ashkenazi Jewish family with gaucher disease in three successive generationsAmerican Journal of Medical Genetics, 1990
- Gaucher disease — Norrbottnian type Ocular abnormalitiesActa Ophthalmologica, 1985