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Intra-monocyte Pharmacokinetics of Imiglucerase Supports a Possible Personalized Management of Gaucher Disease Type 1

Archive ouverte | Berger, Juliette | CCSD

International audience. Background and objectives: Intravenous imiglucerase enzyme replacement therapy for Gaucher disease type 1 administered every 2 weeks is at variance with the imiglucerase plasma half-life of a...

A Cross-Sectional Retrospective Study of Non-Splenectomized and Never-Treated Patients with Type 1 Gaucher Disease

Archive ouverte | Serratrice, Christine | CCSD

International audience. Patients with type 1 Gaucher disease (GD1) present thrombocytopenia, anemia, organomegaly, and bone complications. Most experts consider that the less aggressive forms do not require specific...

The French Gaucher's disease registry: clinical characteristics, complications and treatment of 562 patients.

Archive ouverte | Stirnemann, Jérôme | CCSD

International audience. BACKGROUND: Clinical features, complications and treatments of Gaucher's disease (GD), a rare autosomal-recessive disorder due to a confirmed lysosomal enzyme (glucocerebrosidase) deficiency,...

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