Amicus Therapeutics Wants Effective Treatment Options for Rare, Orphan Diseases

Amicus Therapeutics, a well-known biotechnology company, based in New Jersey, wanted to develop effective treatment options for rare, orphan diseases. The firm is in the pipeline of developing treatment solutions for multiple human genetic disorders. Its migalastat, is a drug that is in the final stage of development would help the physicians to treat people who are struggling with Fabry disease, based on their genetic diagnosis. Amicus Therapeutics confirmed that migalastat is moving to commercial launch stage. SD-101 is another medication that is also in the late-stage of development which would be the first-to-market therapy to address Epidermolysis Bullosa, an unusual genetic connective tissue disorder. Learn more about job offerings of Amicus Therapeutics at Indeed.

SD-101 is in the phase three stage of the testing, and it is also expected to be made commercially available anytime soon. Amicus Therapeutics is also designing a biologics program named ATB200/AT2221. It is a specifically designed Pompe disease ERT allowing it to use along with any pharmacological chaperone. The biotechnology firm is also known for using its Chaperone-Advanced Replacement Therapy platform and biologics to develop new enzyme replacement therapies or ERTs for various diseases including Lysosomal Storage Disorders or LSDs, Pompe disease, Farby disease, and more. Apart from these advanced staged programs, Amicus Therapeutics is also developing many other solutions to address other orphan diseases as well.


The biotechnology company was established in the year 2002, and it went for a public listing in 2007. Initially, the company was funded by a number of venture capitalist firms including, Canaan Partners, New Enterprise Associates, and Radius Ventures. Amicus Therapeutics has received multiple grants in the past including The Michael J. Fox Foundation grant that amounts to $500,000 in 2010. Alzheimer’s Drug Discovery Foundation also offered a grant of $210,300 in 2010 to work together with Icahn School of Medicine for supporting pre-clinical works. Amicus Therapeutics also completed some acquisitions recently to expand their expertise in biotechnology and genetics research. Learn more about the company at

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