Amicus Therapeutics

Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the forefront of developing therapies for rare and orphan diseases. The Company has a robust pipeline of novel, first-in-class, small molecules called pharmacological chaperones for the treatment of lysosomal storage diseases (LSDs). These chaperones may offer a dual-treatment approach for Fabry, Pompe, Gaucher and other LSDs. As orally administered monotherapy agents, pharmacological chaperones are designed to bind to, stabilize and increase the activity of a patient’s own misfolded enzyme. In combination with enzyme replacement therapy (ERT), pharmacological chaperones may improve the uptake of the infused enzyme and potentially improve ERT outcomes.

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