DISEASE MANAGEMENT
Miglustat
What is miglustat?
Miglustat is an oral substrate reduction therapy for Gaucher disease.
INTRODUCING
Eliglustat
The introduction of enzyme replacement therapy and substrate reduction therapy has enhanced the management of Gaucher disease.
Eliglustat is a substrate reduction therapy indicated for the oral treatment of patients with Gaucher disease.1 The mechanism of action of eliglustat is presented in Figure 1. For further information on eliglustat, please consult your local prescribing information.
Figure 1.
Mechanism of action of eliglustat1,2
C-ANPROM/INT//7568; Date of preparation: September 2020
1. Sanofi Genzyme. Cerdelga® EU Summary of Product Characteristics. Last updated January 2020.
2. Stirnemann J, Belmatoug N, Camou F, et al. A review of Gaucher disease pathophysiology, clinical presentation and treatments. Int J Mol Sci 2017; 18: 441.
DISEASE MANAGEMENT
Substrate reduction therapy
What substrate reduction therapies are available for Gaucher disease?
Two oral substrate reduction therapies are available for Gaucher disease: miglustat and eliglustat.
DISEASE MANAGEMENT
Enzyme replacement therapy
What enzyme replacement therapies are available for Gaucher disease?
Three long-term enzyme replacement therapies are available for patients with Gaucher disease: imiglucerase, velaglucerase alfa and taliglucerase alfa.
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