Amicus Therapeutics, founded in 2002, is a publicly traded company that is involved in the very important world of biopharmaceutical research. The NASDAQ trading symbol is FOLD and it opened its first office in Cranbury, New Jersey. The main focus of the company is on rare and orphan diseases. Within these diseases, Amicus Therapeutics focuses more on what is known as lysosomal storage disorders. The company concentrates on Chaperone-Advanced replacement therapies and also enzyme replacement therapies. By 2014, Amicus Therapeutics became arguably the most experienced in the industry as far pharmacological chaperones are concerned.

Amicus Therapeutics is in late stage development of migalastat, which is used to treat Fabry Disease. The reason behind the company’s decision to go forward with this type of treatment is because there are around 3000 people in the US alone who have been diagnosed with it. Fabry disease is a relatively rare disorder that is X-linked and inherited which causes lysosomal storage. The symptoms can start showing either at the early stages or later stages of the disease. However, migalastat can only be used on patients that have amenable genetic mutations. When the patient is ready to take it, it is prescribed as an oral medication.

Amicus Therapeutics is a little different compared to many other pharmaceutical companies in that they do not do any of the manufacturing themselves (Twitter). This part of the work is contracted out to other companies. The company recently opened a new research facility in San Diego California. The company has received numerous grants for medical research, such as $ 500,000 grant from the Michael J. Fox Foundation to support studies done along with the David Geffen School of Medicine at UCLA. Among other notable grants the company received was from the Alzheimer’s Drug Discovery Foundation, a sum that totaled $210,300.

The CEO of Amicus Therapeutics is John F. Crawley who took over the helm back in 2005. In 2013, he headed the takeover of a rival pharmaceutical company Callidus Biopharma. Through this takeover, Amicus got access to the intellectual property rights to enzyme replacement therapy to treat Pompe disease.

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