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Amicus Therapeutics Leads The Way In Rare And Orphan Disease Research

Amicus Therapeutics is a leading biotechnology company that supports treatment for rare diseases worldwide. The company’s mission is to research and produce pharmaceuticals to treat rare and orphan diseases known as lysosomal storage disorders. Amicus Therapeutics was privately held by venture capitalists until 2007 when the company became an IPO and publicly traded on the NASDAQ. Their company headquarters are in Cranbury, New Jersey.


John Crowley is the CEO of Amicus Therapeutics and has held this position since 2010 (MarketWatch). John Crowley is a well-respected biotechnology entrepreneur, and is the co-founder of Novazyme Pharmaceuticals. John’s leadership skills have helped focus the business on patient driven success. Every employee at Amicus Therapeutics is dedicated to the improvement of those born with rare or orphan diseases. Their business model embraces ethics, compliance and integrity in everything all parts of the company.


Amicus Therapeutics is one of a handful of biotechnology companies who are developing drugs for a small sector of the population who are affected by lysosomal storage disorders. The company has several important drugs in production: Migalastat is a drug formulated to treat patients with Fabry disease. Fabry disease is a rare genetic disorder in which the human body cannot release fat due to a lack of enzymes in the body. A second drug in production is SD-101, a treatment for the genetic tissue disorder called Epidermolysis Bullosa. Amicus Therapeutics focus is on researching the effectiveness of novel enzyme replacement therapies and biologics,which are genetically engineered proteins processed from human genes.


Orphan diseases are classified by the USDA as diseases that affect less than 200,000 people in the United States (GoogleFinance). Due to the nature of rare and orphan diseases research in this sector has been historically underfunded. Amicus Therapeutics received a financial helping hand in 2010 from the Michael J. Fox Foundation in the form of a grant for $500,000. In the same year Amicus Therapeutics was granted $210,300 from the Alzheimer’s Drug Discovery Foundation. Amicus Therapeutics purchased the biotechnology company Callidus Biopharma in 2013, and Scioderm in 2015. The company continues to grow as they work towards important biotechnology therapies.

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