The inspiring and unique fact about Amicus Therapeutics and their pursuits is that they global biotechnology company that is focused on rare and orphan diseases. Rare diseases, of course, are those diseases which affect a small percentage of the overall population. Orphan diseases are types of rare diseases whose rarity status means there is not enough funding to find treatments for them.
More common diseases receive endless amounts of funding and research and in many cases, these diseases find cures and/or treatments (ReleaseFact). However, rare diseases because of their minority status receive less funding and attention and therefore often these diseases and those that are the ill-fated infected recipients are left without cures and/or treatment. Amicus Therapeutics has a product development focus on enzyme replacement therapy. Enzyme replacement therapy is a type of treatment whose aim to replace an enzyme that is lacking in the body (https://www.dialdish.com/amicus-therapeutics-fabry-disease/). Amicus Therapeutics is based in Cranbury, New Jersey and several years ago opened a second site in San Diego, California. The company became a publicly traded company in 2007 being traded under the symbol FOLD. Amicus Therapeutics has many positive patient stories. For example, one man who entered the hospital with severe breathing difficulty and was afterward misdiagnosed with Polymyositis.
Subsequently, he was correctly diagnosed with a rare disease known as Pompe. This patient became the first in the world with his condition to receive a diaphragm pacing system which allows him to breathe on his own and return to his family. Such patients and their unique stories and the support they receive from Amicus Therapeutics are beacons of light which help to illuminate a dark sea of mystery and fear, and shine hope so that others may find a brighter tomorrow.