Amicus Therapeutics
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[edit] Background
Amicus Therapeutics is a biopharmaceutical company developing oral therapeutics known as pharmacological chaperones for the treatment of a range of human genetic diseases. Pharmacological chaperone technology involves the use of small molecules that selectively bind to and stabilize proteins in cells, leading to improved protein folding and trafficking, and increased activity. Amicus is initially targeting lysosomal storage disorders, which are severe, chronic genetic diseases with unmet medical needs. Amicus has completed Phase 2 clinical trials of Amigal for the treatment of Fabry disease and is conducting Phase 2 clinical trials of Plicera(TM) for the treatment of Gaucher disease and AT2220 for the treatment of Pompe disease. As a company, they believe that we must constantly build momentum in the fight against human genetic diseases, and are collaborating with advocacy groups.
[edit] Website
http://www.amicustherapeutics.com/
[edit] Recommended Sites
[edit] Links to Other Organizations
* Advocacy Organizations * Academic Institutions * Biotech Companies * Government Agencies * Genetic Alliance * Laboratories * Law/Consulting Firm * Patient Registries * Policy Organizations/Think Tanks * Professional Societies * Other
