Amicus Therapeutics and Fabry Disease

Amicus Therapeutics, Inc. is an international biotechnology company dedicated to the treatment of orphan and rare diseases. The company has modern medicines which have been approved by the World Health Organization. One of its leading products includes migalastat which is a personalized medicine is in its final development stages (Crunchbase).


The medicine will be vital in treating patients with Fabry disease depending on their genetic diagnosis. In addition, there is also another product that Amicus Therapeutic intends to launch very soon, the SD-101. The drug will be the first type of medicine to treat Epidermolysis, a rare genetic connective tissue disorder.


Amicus therapeutic is business and science-driven with a goal of ensuring that patients receive the best kind of treatment. Their product pipeline position platform basically gives the company an edge in developing therapies for rare and orphan diseases. Amicus therapeutic uses the pipeline platform to research on the best ways to reduce pain from patients suffering from uncommon diseases in our society.


What is Fabry Disease?


Amicus therapeutic has dedicated most of its resources to fighting Fabry disease. But what is Fabry disease? Fabry disease is an X-linked disease resulting from mutations from the GLA gene which then encodes the alpha-Gal A enzyme. The mutations can cause the alpha-Gal A to be deficient or absolutely absent. Symptoms of Fabry disease can be severe. They usually start to appear at an early stage but vary from one patient to the other.


The disease causes irreversible, progressive as well as organ damage which involves the cardiac, nervous, renal systems and several other multiple tissues. As a result of the complications, Fabry disease requires lifelong medication.


Amicus therapeutic has put in place several measures to ensure that patients are diagnosed early in enough and placed under treatment. The complications require that a patient stays close to a doctor for monitoring.

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