Amicus Releases Trial Results for Fabry Disease Compound
Amicus Therapeutics reported six-month clinical trial results this week for its lead compound for Fabry disease. The hit the company's share price took this week is an unfortunate example of the uncertainty that comes with investing in emerging biotechnology companies.
Fabry disease is caused by a genetic mutation that causes cells to produce misfolded versions of an important enzyme called alpha-galactosidase A, used to break down proteins and fats. Since they are unstable, they do not arrive at a cell's lysosome to do their work in sufficient quantity. This leads to a buildup of a protein called GL3, which causes serious health problems.
Amicus' drug, migalastat, is designed to stabilize and chaperone these misfolded proteins on their way to lysosomes in order to improve their function.
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