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- Amicus Therapeutics, Inc.
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Overview
Company Summary
Amicus Therapeutics, Inc. is a biotechnology company that primarily focuses on the development of advanced therapies for rare and orphan diseases. The company aims to provide treatment options for patients suffering from devastating genetic disorders.
Amicus Therapeutics utilizes its scientific expertise and innovative technologies to develop precision medicines that can address the underlying causes of various rare diseases. Their approach involves targeting specific enzymes or proteins in the body to restore their normal functioning, thus potentially offering new treatment possibilities.
The company's research and development efforts mainly center around three main therapeutic areas: lysosomal storage disorders, genetic connective tissue disorders, and neurodegenerative diseases. These conditions are often characterized by a lack of treatment options, limited understanding, and significant unmet medical needs.
Amicus Therapeutics is particularly known for its development of enzyme replacement therapies (ERTs) and pharmacological chaperone therapies (PCTs). ERTs involve replacing or supplementing deficient enzymes in patients with genetic disorders. PCTs, on the other hand, use small molecules to stabilize or enhance the activity of defective proteins, enabling them to function more effectively.
The company has a range of potential therapies in various stages of development and clinical trials. These therapies aim to treat rare diseases such as Fabry disease, Pompe disease, and other disorders caused by enzyme deficiencies.
Additionally, Amicus Therapeutics collaborates with patient communities, academic institutions, and other biopharmaceutical companies to advance its mission of delivering groundbreaking treatments. The company is committed to improving the lives of patients with rare diseases by providing innovative therapies that address the root causes of their conditions.