enzimkompenzációs
Enzimkompenzációs, also known as enzyme replacement therapy (ERT), is a medical treatment used to address genetic disorders where the body produces insufficient amounts of specific enzymes. These enzyme deficiencies can lead to the buildup of toxic substances within cells, causing a range of symptoms and progressive damage to various organs. ERT aims to counteract this by administering a functional version of the missing or defective enzyme, typically intravenously.
The goal of enzyme replacement therapy is to reduce the accumulation of substrate molecules that would otherwise
ERT is currently available for a number of rare genetic conditions, including Gaucher disease, Fabry disease,