Category Archives: Fabry Disease

Amicus Therapeutics (Frontier for Advanced Therapeutics)

Amicus Therapeutics a biopharmaceutical company at the forefront of advanced therapeutic treatments. Located in the Garden State of Cranbury, NJ but also has locations in the United Kingdom, France, Germany, Italy, The Netherlands and Spain. The Company went public in (NASADQ)2007 under the trade name FOLD. Before going public the company was sponsored of various capital ventures such as Radius Ventures, and Canaan Partners.)

Amicus Therapeutics specializes in treatments of rare orphan diseases such as Lysosomal Storage Disorders, Epidermolysis Bullosa, and CDKL5. According to the FDA orphan diseases are: medical conditions that affect less than 200,000 people in the USA. Amicus Therapeutics main treatment migalastat(trade name Galafold) is an oral chaperone treatment used to treat Fabry disease. The overall goal of this treatment is to stabilize endogenous mutant enzymes.


John F. Crowley took on the role of CEO of Amicus Therapeutics in 2005. It was under his leadership chaperone treatment migalastat was developed. In 2010 Crowley took on the role as company chairman of the board. Under his leadership Amicus Therapeutics has grown in stock (GoogleFinance). In 2013 the company acquired Callidus Biopharma and in 2015 they acquired Scioderm for up to $947 million in cash and stock. The company received a grant from the Michael J. Fox Foundation in support of studies with the David Geffen School of Medicine, in 2010 they also received a grant from the Alzheimer’s Drug Discovery Foundation to support pre-clinical work in collaboration with Icahn School of Medicine at the Mount Sinai through the Alzheimer’s Disease Research Center. Crowley prides himself on working closely with patients in the research and development process.” We are unique in that our scientists often work hand-in-hand with patient advisors when researching and discovering new approaches to complex diseases for people living with a variety of rare and difficult-to-treat conditions.”(John F. Crowley)

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