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美國Amicus Therapeutics  
Amicus治療是口服的，稱為小分子藥物的藥理伴侶，  在第一級的方法來治療范圍廣泛，包括溶酶體貯積癥的疾病和??神經(jīng)退行性疾病。

Amicus Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of a new class of small molecule, orally-administered drugs to treat a range of human genetic diseases.

Amicus’ innovative therapies apply to conditions in which crucial proteins are defective as a result of improper folding.

Instead of trying to replace these complex proteins, Amicus’ approach uses pharmacological chaperones to selectively bind to the misfolded target protein, increase stability, and help the protein fold into its proper three-dimensional shape. This allows the protein to be trafficked to the specific location in the cell, where it performs its intended biological function. Pharmacological chaperone technology represents a novel, next-generation approach to the management of human genetic diseases and offers the potential to improve treatment options for patients.

Founded in 2002, Amicus has assembled an outstanding leadership team that includes scientific, medical, regulatory, and business professionals with successful track records of developing and bringing to market drugs for rare genetic diseases. Amicus continues to develop its pharmacological chaperone technology and has built a significant intellectual property portfolio, as well as a robust clinical and preclinical pipeline of small molecule, orally-administered agents.

Amicus’ most advanced product candidate, AT1001, completed Phase 2 clinical trials for Fabry disease in December 2007. For information about the positive results from these trials, click here. Based on these results, Amicus plans to initiate a Phase 3 clinical trial of AT1001.

The company’s second most advanced clinical product candidate, AT2101, is in Phase 2 clinical trials for Gaucher disease. One study was completed in March, 2008 and additional studies are ongoing and planned. For information about the positive results of the first trial, click here. To learn more about ongoing trials, click here.

Amicus’ third most advanced product candidate, AT2220 for the treatment of Pompe disease, completed Phase 1 clinical trials in October 2007. For information about the positive results from these trials, click here. Based on these results, Amicus plans to initiate a Phase 2 clinical trial of AT2220.

Additionally, Amicus’ biologists and medicinal chemists are leveraging the company’s breakthrough scientific platform to conduct targeted drug development, allowing for the rapid identification and optimization of lead compounds across a variety of disease areas, including Parkinson's Disease.

Amicus has broad, issued intellectual property around the methods, use, and composition of pharmacological chaperones to treat genetic diseases. In addition to the currently issued patents, Amicus has several patent applications pending.

Amicus completed an Initial Public Offering in May 2007 and entered into a strategic collaboration with Shire Human Genetic Therapies in November 2007.

Amicus Therapeutics' headquarters and laboratories are located in Cranbury, NJ.

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