An open-label, noncomparative study of miglustat in type I Gaucher disease: Efficacy and tolerability over 24 months of treatment
Author:
Publisher
Elsevier BV
Subject
Pharmacology (medical),Pharmacology
Reference45 articles.
1. Gaucher's disease: Clinical features and natural history;Cox;Baillieres Clin Haematol,1997
2. Gaucher disease. Clinical, laboratory, radiologic, and genetic features of 53 patients;Zimran;Medicine (Baltimore),1992
3. Genetics of the sphingolipidoses;Knudson,1962
4. Twin pairs showing discordance of phenotype in adult Gaucher's disease;Lachmann;QJM,2004
5. Replacement therapy for inherited enzyme deficiency—macrophagetargeted glucocerebrosidase for Gaucher's disease;Barton;N Engl J Med,1991
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