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Recombinant enzyme used to treat Pompe disease; Lysosomal Alpha Glucosidase
Recombinant enzyme used to treat Pompe disease; Lysosomal Alpha Glucosidase
Lysosomal alpha glucosidase is an enzyme used to treat Pompe disease (lysosomal storage disorder).

The enzyme lysosomal alpha glucosidase is used to treat Pompe illness (lysosomal storage disorder). It is a hereditary condition in which the body's cells accumulate a complex substance called glycogen. The lack or impairment of an enzyme termed acid alfa glucosidase, which breaks down complex sugars in the body, is a symptom of Pompe disease. The liver, heart, and muscles are the most commonly affected organs. It can affect anyone, but it is more common among African-Americans and some Asian communities.

In January 2021, Sanofi filed avalglucosidase alfa in Japan. The only effective treatment for Pompe illness is ERT. The procedure entails injecting lysosomal alpha glucosidase into the bloodstream. As a result, the body depletes glycogen stores and protects cells against toxic accumulation. The hydrolysis of starch to simple sugars is catalysed by glucosidase enzymes. These enzymes aid in the digestion of dietary carbohydrates and starches in humans, resulting in the production of glucose for intestinal absorption and an increase in blood sugar levels.

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