Amicus Therapeutics Pioneering Studies to Treat Rare Orphan and Genetic Diseases

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A company that went public under the NASDAQ trading symbol FOLD in 20017, Amicus Therapeutics is an American biopharmaceutical company with its international headquarters in Britain, while the corporate headquarters in Cranbury, NJ. By being passionate about what they do, the company is a global leader at the apex of advanced and innovative therapies that treat several orphan diseases that are devastating and rare.



Amicus Therapeutics boasts is famous for having an active development chain for treating several human genetic diseases. It was funded by many firms dealing in venture capital such as New Enterprises, Canaan Partners, and Radius Ventures. Some of the rare disorders that Amicus Therapeutics specializes in handling are collectively known as lysosomal storage disorders.



Noted in the pharmaceutical industry for having the broadest range of molecule pharmacological chaperones in 2014, the product development of Amicus Therapeutics is concentrated on the development of enzyme replacement therapies or ERTs and it is largely based on Chaperone-Advanced Replacement Therapy or CHART platform technologies for the treatment of Pompe disease, Fabry disease, and some other Lysosomal Storage Disorders or LSDs.



The leading test product that Amicus Therapeutics has is a personalized medication called migalastat which is in mature stage development. It will be suitable for treating people suffering from Fabry disease depending on the diagnosis of their genes ( Another product by Amicus Therapeutics which is also in the mature stage development is known as SD-101. It will be used to treat a disorder of tissue connection called Epidermolysis Bullosa or EB as a first-to-market therapy.



Presently the leading biological therapy program by Amicus Therapeutics is a form of therapy which is a uniquely produced therapy for Pompe infection which will be used when combined with other controlling drugs. The company has received grants amounting to US$500,000 from The Michael J. Fox Foundation to help coordinate and support joint studies conducted in partnership with other leading medical institutions (Marketwatch). It is also pioneering other studies to find cures for Alzheimer’s disease. Sometimes in November 2013 Amicus Therapeutics obtained intellectual property and proprietary material for therapy treatment of Pompe disease.