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AHU-377 is the development code for Ivanglucerase, a pharmaceutical compound used in the treatment of Gaucher disease. Gaucher disease is a rare genetic disorder caused by a deficiency of the enzyme glucocerebrosidase, which leads to the accumulation of fatty substances (lipids) in various organs, such as the liver, spleen, and bone marrow.
Ivanglucerase (AHU-377) is an enzyme replacement therapy (ERT) designed to replace the missing or deficient enzyme, helping to reduce the buildup of glucocerebroside and alleviate the symptoms of Gaucher disease. It is a recombinant version of the enzyme that is administered through an intravenous infusion.
The drug was developed by Ahus, a biotechnology company, and is one of the alternatives to other approved treatments for Gaucher disease, such as imiglucerase and velaglucerase alfa.
AHU-377 (also known as Ivanglucerase) is primarily used in the treatment of Gaucher disease, a rare genetic disorder. The application of AHU-377 focuses on enzyme replacement therapy (ERT) to manage and alleviate the symptoms of this disease. Here’s how it is applied:
Gaucher Disease Type 1:
AHU-377 is used to treat Type 1 Gaucher disease, the most common form of the disorder, which is characterized by a deficiency of the enzyme glucocerebrosidase. This deficiency leads to the accumulation of a fatty substance called glucocerebroside in cells of the liver, spleen, bone marrow, and lungs, causing damage to these organs.
The treatment aims to replace the missing enzyme and reduce the buildup of glucocerebroside, thus improving the function of affected organs and alleviating symptoms like organ enlargement (splenomegaly and hepatomegaly), bone pain, anemia, and fatigue.
Bone Health:
One of the prominent features of Gaucher disease is bone involvement, including osteopenia, fractures, and bone pain. AHU-377 helps to reduce the accumulation of glucocerebroside in bone marrow, improving bone health and reducing the risk of fractures.
Enzyme Replacement Therapy (ERT):
AHU-377 works by delivering a recombinant version of glucocerebrosidase (the enzyme missing in Gaucher disease) into the patient’s body through intravenous (IV) infusion. This replaces the deficient enzyme and helps in the breakdown of glucocerebroside, alleviating the symptoms and preventing further organ damage.
Long-term Management:
Enzyme replacement therapy like AHU-377 is typically a lifelong treatment for patients with Gaucher disease. Regular infusions help maintain normal enzyme levels and prevent the progression of disease-related symptoms, especially in those with moderate to severe disease.
Improved organ function (such as the liver and spleen) through reduction in organ enlargement.
Improved blood counts, including hemoglobin and platelet levels.
Reduced bone pain and risk of fractures through better bone health.
Overall improvement in quality of life by addressing both physical symptoms and complications associated with the disease.
AHU-377 is used specifically for managing Gaucher disease, offering patients a treatment option to address the underlying enzymatic deficiency, control symptoms, and improve long-term health outcomes.
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