Amicus Therapeutics is an American biopharmaceutical company based in New Jersey and established in 2002. The role and focus of Amicus Therapeutics are to find treatments or cures for very rare and orphan diseases or illnesses. These rare and orphan diseases have specific meaning in the medical world because these diseases affect only a particular number of people in a population. Specifically, research has shown that rare diseases are genetic, chronic, and in some instances are seen as incurable. And this is where the expertise and hard work of Amicus Therapeutics come into play (Twitter). It is their mission to eventually eradicate these life-threatening genetic diseases and give the highest quality of support to all in constant suffering from these diseases. In a time where healthcare can be challenging, Amicus Therapeutics demonstrates that is imperative to have a passionate and cohesive stance on every aspect of their work.
Amicus Therapeutics stresses the importance of engaging the needs of the patient. Not only does Amicus Therapeutics spend time with the patients to make sure their healthcare needs are being met but Amicus Therapeutics also honors the needs of the families or caregivers of their patients as well. The persistence of Amicus Therapeutics really shows in how they approach their treatment specifications regarding the programs and clinical studies for diseases such as Fabry Disease, Epidermolysis Bullosa Disease, and Pompe Disease. These are all seen as quite devastating rare and orphan diseases due to the intense, rare, progressive, genetic, and chronic condition of each disease. Amicus Therapeutics takes their skills into account when developing the best possible treatment of these diseases and so many others. At the end of the day, Amicus Therapeutics have established themselves as a force to be reckoned with in the conquering of rare and orphan diseases. The importance of bringing patients the best possible therapeutic treatment and answers to diseases not very well-known or that do not affect very large populations, is beyond precedent.