title: [Course of a case of Gaucher's disease type 1 treated over a year with glucocerebrosidase (Cérédase)].
abstract: Gaucher's disease is caused by a deficiency of glucocerebrosidase. Enzyme replacement therapy with glucocerebrosidase (Ceredase) is available in France since 1991. We report the clinical effectiveness in a woman with type 1 Gaucher's disease treated with the enzyme for one year. Hepatomegaly regressed, bone pain decreased but improvement of severe skeletal manifestations were slow and incomplete.
authors.affiliation: Service de médecine interne du Pr C Carbon, Paris.
authors.forename: N
authors.initials: N
authors.lastname: Belmatoug
authors.forename: M
authors.initials: M
authors.lastname: Azorin
authors.forename: D
authors.initials: D
authors.lastname: Huten
authors.forename: T B
authors.initials: TB
authors.lastname: de Villemeur
authors.forename: C
authors.initials: C
authors.lastname: Carbon
mesh_terms: D005260:Female; D005776:Gaucher Disease / Q000188:drug therapy*; D005962:Glucosylceramidase / Q000627:therapeutic use*; D006801:Humans; D008875:Middle Aged
chemical_list: D005962:Glucosylceramidase
vernacular_title: Evolution d'une maladie de Gaucher de type 1 traitée pendant un an par la glucocerébrosidase (Cérédase).