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Amicus Therapeutics Journey to Developing Cure for Rare Diseases


Amicus Therapeutics is a biotechnology company that began in 2002 and has its international headquarters in Buckinghamshire in the United Kingdom and corporate headquarters in Cranbury. The company has its presence in other countries like France, Spain, Netherlands Germany and Italy. In 2007 under the leadership of the CEO John Crowley the company sold its shareholding to the public and started trading on NASDAQ.


Amicus Therapeutics is at the forefront of treating rare and orphan disease using advanced therapies. The company has unique technology and medicine used in treating diseases like Lysosomal storage disorders, Fabry, Pompe and genetic connective skin disorders. The company endeavors to make a difference in the lives of patients with these diseases, which in turn transforms the lives of many families.


Amicus Therapeutics is developing many products, and one of their primary product is migalastat which is used to treat Fabry disease from genetic diagnosis. They also have a product known as SD-101 that is in the late development stage and will be used to treat Epidermolysis Bullosa (EB).


The company is using a very innovative technology platform to develop their treatments. The company has dedicated scientists who find enhanced treatment for the patients. They are developing a pharmacological chaperone technology that will be used to treat lysosomal storage disorder which results from mutation of some lysosomal enzymes. They are also developing an enzyme technology to assist patients with LSD’s (https://www.dialdish.com/amicus-therapeutics-fabry-disease/). The company largely use the chaperone-advanced replacement therapy to generate most of its product. Amicus has a team dedicated towards invention and ensuring that their patients get better and better.


Amicus therapeutics partners with the patient organization, patients, caregivers, and doctors to support the community that has people with these rare diseases (ForexWizard). They have come up with initiatives like the patient advisory board to make the lives of the patient better by listening to them, coming up with better therapies and providing resources that are of help to the community. The organization is also involved in charitable giving more so towards education, advocacy and to those affected by the disease that the company focuses on.


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