Druggio Logo

Druggio

Gaucher disease

Gaucher disease is an autosomal recessive lysosomal storage disorder caused by deficient beta-glucocerebrosidase (glucosylceramidase) activity or saposin C which is an activator of beta-glucocerebrosidase in sphingolipid metabolism. The enzymatic defects lead to the accumulation of glucosylceramide (GC) in lysosomes of affected cells. Despite the fact that Gaucher Disease consists of a phenotype, with varying degrees of severity, it has been sub-divided in three subtypes according to the presence or absence of neurological involvement. The sub-types are Type 1, 2 and 3.

Drugs that treat Gaucher disease

Cerdelga

Made by

Sanofi

Approval date

2015/3/26

Vpriv

Made by

Takeda

Approval date

2014/7/4