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References
- Outcome of 27 patients with Hurler's syndrome transplanted from either related or unrelated haematopoietic stem cell sources.Bone Marrow Transplant. 2003; 31: 1105-1117
- Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: Results of a multinational study of recombinant human alpha-l-iduronidase (laronidase).Pediatrics. 2007; 120: e37-e46
- Urinary globotriaosylceramide excretion correlates with the genotype in children and adults with Fabry disease.Mol Genet Metab. 2008; 93: 331-340
PaschkeE, FaulerG, UrbanW, et al. Isoforms of globotriaosylceramide in urine can be used to identify female Fabry patients. Lecture presented at: 16th ESGLD Workshop-European Study Group of Lysosomal Diseases, September 27-30, 2007, Perugia, Italy.
- Influence of antibody formation on reduction of globotriaosylceramide (GL-3) in urine from Fabry patients during agalsidase beta therapy.Mol Genet Metab. 2007; 92: 271-273
- Glucose tetrasaccharide as a biomarker for monitoring the therapeutic response to enzyme replacement therapy for Pompe disease.Mol Genet Metab. 2005; 85: 247-254
- The natural course of infantile Pompe's disease: 20 Original cases compared with 133 cases from the literature.Pediatrics. 2003; 112: 332-340
- Plasma and metabolic abnormalities in Gaucher's disease.Baillieres Clin Haematol. 1997; 10: 691-709
- Clinical evaluation of chemokine and enzymatic biomarkers of Gaucher disease.Blood Cells Mol Dis. 2005; 35: 259-267
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© 2008 Excerpta Medica Inc. Published by Elsevier Inc.
