The Amicus Therapeutics (FOLD) pipeline consists of novel, first-in-class treatments for a range of genetic diseases. The most advanced compounds along its pipeline address the largely unmet needs of patients with lysosomal storage disorders ((LSDs)).
Lysosomes, if you are having trouble remembering, are the cell's waste disposal system. They engulf and break down worn out organelles, bacteria, viruses, and play a large role in food digestion. LSD causes vary, but most often deficiency in a specific enzyme function leads to a buildup of debris, almost like a city with garbage collectors on strike.
Amicus uses pharmacological chaperone technology to develop small molecules, or chaperones that selectively binds to LSD specific target proteins, keeping them folded in the correct,...
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