Amicus Therapeutics operates out of Canbury, New Jersey and has been publicly traded since 2007. Though considerably small compared to most biopharmaceutical companies, Amicus Therapeutics has created a development pipeline of first in class treatments for people with rare genetic disorders.
The main mission of Amicus Therapeutics is to research and treat people suffering from various lysosomal storage disorders. These disorders, often abbreviated as ‘LSD’, compromise of a group of at least 50 distinct genetic diseases such as Fabry Disease, Epidermolysis Bullosa, and Pompe Disease. New types of lysosomal storage disorders are being discovered as more research is done on various genetic conditions in order to biochemically characterize them.
Amicus Therapeutics has developed various medicines that help patients suffering from these diseases. Migalastat, also known as Galafold, is a long term treatment for adults and adolescents with Fabry disease with a unique mutation of the alpha galactosidase enzyme.
The CEO of Amicus Therapeutics is John F. Crowley. Crowley began is work in the biopharmaceutical industry shortly after his own two children were diagnosed with Pompe’s disease in 1998. His first job in the field was with Bristol-Myers Squibb where he immersed himself in health research and raised money for Pompe awareness. When Crowley realized that even though various universities around the world were doing research on these diseases they weren’t communicating with one another because of the competition in the academic world. Frustrated, he decided to turn his professional focus on searching out and creating treatments for Pompe disease. This passion would eventually lead him to his current position at Amicus Therapeutics.
Amicus Therapeutics prides itself on it’s team of resilient and persistent people dedicated into making medicines that will meaningfully improve the lives of their patients (Facebook). With an already impressive success record of treatments available in the market and a CEO with a personal mission to cure these diseases, it isn’t alarming that Amicus Therapeutics is, and will remain, at the forefront of scientific research for LSD’s.