Category Archives : Rare Disease

Home  >>  Rare Disease

How Amicus Therapeutics Treats Rare And Orphan Diseases



Amicus Therapeutics, Inc. is a publicly traded company that is headquartered in New Jersey. The goal of this company is to create therapies for rare and orphan diseases that afflict people around the world. In particular, they are focused on treating human genetic diseases.

The lead product candidate at Amicus Therapeutics is Migalastat which has been designed as a therapy for Fabry disease. Another drug in the later stages of research is SD-101 which would be the very first drug therapy for Epidermolysis Bulluso which is a rare genetic disease that negatively affects connective tissue in the afflicted person’s body. Visit Google Finance to know more about Amicus Therapeutics.

The Chairman of the Board and Chief Executive Officer of Amicus Therapeutics is John F. Crowley. Other top leaders of the company include Bradley L. Campbell who serves as the President and Chief Operating Officer and Chief Financial Advisor Chip Baird. Overall, the leadership group at Amicus Therapeutics has years of experience in the pharmaceutical industry and ably lead the company on its mission.


There are currently three main diseases that Amicus Therapeutics has drugs under research for. This includes the aforementioned Fabry disease and Epidermolysis Bulloso. They also are researching other drugs to treat Pompe disease which is caused by too much sugar building up in the person and thereby damaging their heart, liver, and muscles throughout the body.

It was in 2007 that Amicus Therapeutics had their initial public offering. They trade under the system FOLD on the NASDAQ listing. Before the company went public it had been supplied capital by a number of venture capitalist companies. This included, among others, Canaan Partners and Radius Ventures. Much of the research performed at Amicus Therapeutics is done through collaboration agreements with other companies in the industry such as JCR Pharmaceutical and GlaxoSmithKline. Visit to know more about Amicus Therapeutics.