Amicus Therapeutics: The Rare Company Taking Care of Rare Patients

Most hospitals around the globe have dedicated their resources to research and treatment of the acute diseases like cancer, heart diseases, and HIV among others. In a world where major diseases claim lives of many on a daily basis, the few and rare genetic disorders are almost ignored or forgotten. This is not the case for one company, Amicus Therapeutics, which is based in Cranbury, New Jersey ( Amicus Therapeutics specializes in biopharmaceuticals to research and come up with drug and therapies that treat rare genetic conditions affecting people in the society.


Amicus Therapeutics was founded back in 2002 and went public in the year 2007 (YahooFinance). As at 2013, the company had 92 employees and was run by John Crowley as the CEO, Bradley Campbell as the COO and William Braid as the CFO. Although Amicus Therapeutics has no manufacturing capabilities, it relies on contract manufacturers who have worked for hand in hand with the company. It was through this good working relationship that Amicus Therapeutics extended its operations to San Diego, where the company opened a second research site in 2008.


Amicus Therapeutics deals with rare and orphan conditions like Lysosomal storage Disorders, and Pompe and Fabry disease among other conditions. The company has had great success with treatment options. Some of their candidate products is the Migalastat, which is a personalized medicine used to treat people with Fabry disease depending on their diagnosis. SD-101 is yet another product that is in its late stage of development. SD-101 is aimed at treating a rare connective tissue disorder, Epidermolysis Bullosa (EB.


Amicus Therapeutics aims at developing novel enzyme replacement therapy (ERT) to treat the above-mentioned conditions. Through biotechnology, engineering, and research, Amicus Therapeutics develops its products based on a Chaperone-Advanced Replacement Therapy.


People from all over the world have suffered from rare genetic conditions that have often been misdiagnosed. This is the main reason why Amicus Therapeutics is working hard every day to offer lasting solutions.