Home > Drugs A – Z > Taliglucerase Alfa (Intravenous route)

Taliglucerase Alfa (Intravenous route)

Taliglucerase alfa injection is used for the long-term treatment of type 1 Gaucher disease. This disease is caused by the lack of a certain enzyme in the body called glucocerebrosidase. This enzyme is necessary for the body to use fats correctly, and fats will build up in certain areas of the body if the enzyme is not present. Taliglucerase alfa replaces the missing enzyme to help the body process fats.

What works?

Learn more about the effects of these drugs. The most reliable research is summed up for you in our featured article.

. … Read more
Brand names include
Elelyso
Other forms
By injection
Drug classes About this
Enzyme, Enzyme Replacement

What works? Research summarized

Evidence reviews

Treatment options for Gaucher disease

Gaucher disease, a rare disorder, is caused by inherited deficiency of the enzyme glucocerebrosidase. This defect leads to the build‐up of a fatty material called glucocerebroside in various cells in the body. Untreated individuals may suffer from anaemia, a decrease in platelet counts, massive enlargement of the liver and spleen, and damage to the bones. Two different types of treatment are available: the intravenous supplementation of the deficient protein glucocerebrosidase (enzyme replacement therapy), or the oral administration of a drug that slows down the production of the fatty material that it normally breaks down (substrate reduction therapy).

Enzyme-Replacement Therapies for Lysosomal Storage Diseases [Internet]

Lysosomal storage diseases (LSDs) comprise about 50 unique monogenic autosomal or X-linked diseases with an estimated combined incidence of 1 in 7,000 to 8,000 live births. They occur secondary to genetic mutations that result in deficiency or reduced activity of native intracellular enzymes that catabolize biological macromolecules. These enzyme defects result in accumulation of specific macromolecular compounds within lysosomes in various tissues and organs, causing progressive damage that can become life-threatening in some diseases. LSD management traditionally involved supportive care measures tailored to disease stage, the organs and systems involved, and the degree of impairment. However, enzyme-replacement therapy (ERT) is now commercially available for six LSDs, typically used lifelong with traditional management practices for each.

Summaries for consumers

Treatment options for Gaucher disease

Gaucher disease, a rare disorder, is caused by inherited deficiency of the enzyme glucocerebrosidase. This defect leads to the build‐up of a fatty material called glucocerebroside in various cells in the body. Untreated individuals may suffer from anaemia, a decrease in platelet counts, massive enlargement of the liver and spleen, and damage to the bones. Two different types of treatment are available: the intravenous supplementation of the deficient protein glucocerebrosidase (enzyme replacement therapy), or the oral administration of a drug that slows down the production of the fatty material that it normally breaks down (substrate reduction therapy).

PubMed Health Blog...

read all...