The Excellent Contributions of Amicus Therapeutics to the Pharmaceuticals Industry

Amicus Therapeutics Inc. is an internationally recognized biotechnology firm that has specialized in developing advanced therapies that can be used in curing a variety of life-threatening rare and orphan diseases. The company has currently developed several treatments for different human genetic diseases. Amicus’ main offices are located in Cranbury, New Hersey, The firm has been publicly traded since 2007, and it uses a Nasdaq Stock Market trade symbol, FOLD. Its business symbol would have been AMTX, but it was withdrawn after an unsuccessful planned public offing in 2006. Before Amicus’ IPO, the growth of the company depended on funding from venture capital companies such as Canaan Partners, New Enterprise Associates, and Radius Ventures.

The main disorders that Amicus Therapeutics currently deals with are known as lysosomal storage disorders. The company has been utilizing the Chaperone-Advanced Replacement Therapy (CHART®) in developing its products. Most of the cures that it currently creates are enzyme replacement therapies (ERTs). According to a pharmaceuticals industry report that was published in 2014, Amicus Therapeutics is the sector’s leader in the development of small molecule pharmacological chaperones. The firm targets mutated proteins in developing excellent therapies for the various human genetic diseases. Its scientists have been striving to ensure that people who suffer from rare diseases are cured.

The biotechnology company’s primary product candidate is called migalastat. The medicine is personalized and is currently in its latest stage of development. Migalastat will be used in treating people who have Fabry disease after they have undergone genetic diagnosis. Another product candidate of Amicus Therapeutics is SD-101, and it is currently in the late stage of development. SD-101 is a will be the first therapy in the market that will be used in curing Epidermolysis Bullosa (EB), which is a rare genetic disorder. The company is also striving to develop many other enzyme replacement therapies for Pompe disease, Fabry disease, and many other Lysosomal Storage Disorders by using its Chaperone-Advanced Replacement Therapy and biologics technologies. Amicus Therapeutics’ top biologic program is ATB200/AT2221. This plan is an exclusively developed Pompe disease ERT that will be offered to the patients together with a pharmacological chaperone.

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