Enzyme replacement therapy “drug holiday”: Results from an unexpected shortage of an orphan drug supply in Australia
Author:
Publisher
Elsevier BV
Subject
Cell Biology,Hematology,Molecular Biology,Molecular Medicine
Reference8 articles.
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2. Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease;Hollak;Blood Cells Mol. Dis.,2010
3. The Gaucher registry: demographics and disease characteristics of 1698 patients with GD1;Charrow;Arch. Intern. Med.,2000
4. Spontaneous regression of disease manifestations can occur in type 1 Gaucher disease; results of a retrospective cohort study;Boomsma;Blood Cells Mol. Dis.,2010
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