It is easy to forget that diseases are not few, but many. When we think of major ailments, we think of lung cancer, obesity, and Osteoporosis. These issues bring us images of those in suffering and pain. However “orphan” also exist, ailments affect less than 20,000 recorded sufferers. These disorders are still present…and deadly.
Amicus Therapeutics, Inc. is a pharmaceutical organization dedicated to treating these rare diseases (https://www.crunchbase.com/organization/amicus-therapeutics). Amicus Therapeutics pioneers Chaperone-Advanced Replacement Therapy, which inserts small molecular substances into cells to fix deformed proteins. Drugs Amicus Therapeutics has in development include Migalastat, a treatment for the painful Fabry Kidney disease, SD-101, which targets the skin destroying Epidermolysis Bullosa, and ATB 200/2001, which seeks to save children and adults from lung and muscle issues caused by Pompe disease.
Since its first public offering in 2007, Amicus Therapeutics has met many success. In 2008 it expanded from its headquarters in Cranbury, New Jersey to an additional facility in San Diego. In 2010 The Michael J. Fox Foundation granted $500,000 to Amicus Therapeutics for studies with the David Geffen School of Medicine at UCLA. This proceeded a $210,300 award from the Alzheimer’s Drug Discovery Foundation.
Acquisitions such as Callidus Biopharma in 2013 and Scioderm in 2015 allow Amicus Therapeutics to continue to grow and thrive. Its mission to cure many of the most debilitating small scale diseases and disorders comes closer and closer to fulfillment, and the plight of these patients does not go unnoticed (SeekingAlpha).
The most telling proof of the dedication of Amicus Therapeutics comes from their website itself.
“The needs of patients in the rare disease community are at the center of our inventive science, our commercial organization, and our clinical programs. Our goal throughout all levels of the organization is to make a meaningful difference in the lives of these patients and their caregivers.”
While few may know about orphan diseases, Amicus Therapeutics exists to help those who few seek to cure, and yet still suffer and die before their time.