Browse Category: Pharmaceutical

Amicus Therapeutics, Inc. is not backing down

We hear about cancer, heart disease, and diabetes because so many people suffer from these diseases. However, many diseases that only affect a very, small number of individuals and get much less money for research and treatments. That is where Amicus Therapeutics, Inc. is filling the gap.


Amicus Therapeutics, Inc., located in Cranbury, New Jersey, is a global biotechnology company specializing in developing treatments for rare and orphan genetic diseases ( A rare disease only affects a small percentage of the total population, but an orphan disease is much rarer and affects such a small number of individuals that there is not enough support for its research and development treatments. Amicus Therapeutics, Inc. and its team are developing treatments for such diseases, particularly lysosomal storage disorders, in which certain enzymes are missing within the cell causing disruption of cell function or its death. These diseases affect people of all ages, compromising their quality of life and often resulting in death. Amicus Therapeutics, Inc. is actively pursuing solutions and treatment.


One of the solutions that is in development is migalastat. This product will be used to fight Fabry disease, in which sphingolipids within the cell do not metabolize properly. Another product is SD-101, which will treat Epidermolysis Bullosa, a genetic connective tissue disorder. As certain enzymes are missing in many of the lysosomal storage disorders, Amicus Therapeutics, Inc, is also working to find enzyme replacements to treat these diseases.


CEO, John F. Crowley is passionate in what the company is doing. By making decisions as if they, their child, or a family member were living with this disease changes puts a different perspective on what the company does and on how the team works and thinks.



The entire Amicus Therapeutics, Inc. team cares and is committed to providing solutions for individuals suffering from these diseases and for their families ( It is their desire to provide therapies as quickly as possible by working hard, being innovative, and sometimes thinking outside the box. It is a fight, but no one on the Amicus Therapeutics Inc. team is backing down from it.