entsyymivaihehoito

Ensyymivaihehoito, known in English as enzyme replacement therapy (ERT), is a medical treatment designed to correct a deficiency in certain enzymes. This therapy is primarily used for a group of rare genetic disorders, often called lysosomal storage diseases, where a specific enzyme is either missing or doesn't function correctly. These diseases lead to the buildup of complex molecules within cells, causing progressive damage to various organs and tissues. ERT aims to replenish the missing or deficient enzyme, thereby helping to break down the accumulated substances and alleviate the symptoms of the disease.

The treatment involves administering the replacement enzyme intravenously, typically on a regular schedule, such as weekly or bi-weekly. The enzyme used is often produced through recombinant DNA technology, ensuring a consistent and pure supply. ERT is not a cure for these conditions, but it can significantly slow disease progression, reduce organ enlargement, improve mobility, and enhance the overall quality of life for affected individuals. The effectiveness and specific benefits of ERT can vary depending on the particular enzyme deficiency, the severity of the disease, and the age at which treatment begins. Ongoing research continues to explore new enzymes and improved delivery methods for ERT.