Amicus Therapeutics

About:

Amicus Therapeutics is a biopharmaceutical company developing therapies for the treatment of rare and orphan diseases.

Website: http://www.amicusrx.com

Twitter/X: amicusrx1

Top Investors: New Enterprise Associates, MidCap Financial, Muscular Dystrophy Association, Silicon Valley Bank, Frazier Healthcare Partners

Description:

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.