While SRT and ERT work in different ways, the goal of each treatment is the same: To reduce the accumulation of excess GL-11
Understanding treatment options
More than 90% of patients with Gaucher disease have type 1, making it the most common type of Gaucher disease. Gaucher disease type 1 can be effectively managed.2 Treatment options are available, including oral therapies.
Treating Gaucher disease type 1
There are 2 treatment approaches for Gaucher disease type 11:
- Enzyme replacement therapy (ERT)
- Substrate reduction therapy (SRT)
While SRT and ERT work in different ways, the goal of each treatment is the same: to reduce the accumulation of excess GL-1.
Enzyme replacement therapy (ERT)
ERT works by replacing deficient beta-glucosidase needed to break down excess GL-1.1,3
Substrate reduction therapy (SRT)
SRT inhibits glucosylceramide synthase, thereby slowing down system-wide accumulation of GL-1, allowing the cells’ residual enzyme activity to break down the substrate.1,6
Untreated
Deficient enzymatic catabolism of GL-1 due to Gaucher disease type 1 leads to accumulation of GL-1 in the lysosomes4
.svg)
SRT
SRT specifically inhibits GCS, slowing down the production of GL-1 to reduce accumulation of GL-14
.svg)
ERT
ERT works by replacing deficient beta-glucosidase needed to break down excess GL-15
.svg)
%20(1).svg)
Indication
References: 1. Stirnemann J, Belmatoug N, Camou F, et al. A review of Gaucher disease pathophysiology, clinical presentation and treatments. Int J Mol Sci. 2017;18(2):441. 2. Mistry PK, Cappellini MD, Lukina E, et al. A reappraisal of Gaucher disease-diagnosis and disease management algorithms. Am J Hematol. 2011;86(1):110-115. 3. Deegan PB, Cox TM. Imiglucerase in the treatment of Gaucher disease: a history and perspective. Drug Des Devel Ther. 2012;6:81-106. 4. Cerdelga (eliglustat). Prescribing information. Genzyme Corporation, Cambridge, MA. 5. McEachern KA et al. Mol Genet Metab. 2007;91(3):259-267. 6. Shayman JA. Eliglustat tartrate: glucosylceramide synthase inhibitor treatment of type 1 Gaucher disease. Drugs Future. 2010;35(8):613-620.
