Technology Profits Confidential

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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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Technology Profits Confidential

With new technologies in mobile computing, biotechnology and 3-D printing, it’s nearly impossible to keep up with the throngs of new innovation and still be able to forecast which companies are set to profit. Technology Profits Confidential is your one-stop shop for the companies of tomorrow’s technology… and how you can profit from them.


Ray Blanco

Ray Blanco is the editor of Technology Profits Confidential as well as Breakthrough Technology Alert, FDA Profit Alert, and Technology Profits Daily. Ray has been with Seven Figure Publishing since 2010. In 2019, his closed positions in Technology Profits Confidential outperformed the S&P500 by 50%.

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