title: Enzyme replacement therapy for Gaucher Disease: the only experience in Malaysia.
abstract: Gaucher Disease may now be treated with enzyme replacement therapy (ERT) or bone marrow transplantation (BMT). Both have their advantages and disadvantages. Results with BMT are curative when successful but limited by the scarcity of an appropriate donor. ERT offers very good relief of symptoms but treatment is lifelong and cost of treatment exorbitant. Patients in developing countries are particularly disadvantaged and management remains a dilemma for both doctor and patient.
authors.affiliation: Department of Paediatrics, University of Malaya, 50603, Lembah Pantai, Kuala Lumpur.
authors.forename: L L
authors.initials: LL
authors.lastname: Chan
authors.forename: H P
authors.initials: HP
authors.lastname: Lin
mesh_terms: D002648:Child; D002675:Child, Preschool; D005260:Female; D005776:Gaucher Disease / Q000188:drug therapy*; D005962:Glucosylceramidase / Q000627:therapeutic use*; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008296:Malaysia
chemical_list: C070292:alglucerase; D005962:Glucosylceramidase