Amicus Therapeutics: A Rare Company Targeting Rare Diseases

According to Wikipedia, a rare disease is any disease that affects a small percentage of the population. Many rare diseases are genetic and appear early in life. I’ve heard the question “if rare diseases are so rare, why do they matter?” Well, a global biotechnology company, Amicus Therapeutics, Incorporated believe rare diseases matter. Specifically focusing on mutated proteins, Amicus Therapeutics leverages their Innovative technology platforms on developing treatments for human genetic diseases. Showcasing their robust biotechnology they take bold approaches toward developing treatments to help patients who suffer from rare diseases.


With a solid pipeline of treatments for various human genetic diseases, Amicus Therapeutics is at the forefront of advanced therapies to treat devastating rare and orphan diseases. Listening to and learning from individual patients and organizations, doctors and caregivers, Amicus patient and professional advocates discover how to better provide healing beyond the disease.


Amicus Therapeutics has three clinical programs and the ability to Leverage biologic capabilities and platform technologies to further expand the pipeline for various rare human diseases (WeeklyOpinion). Furthermore, Amicus provides resources and an abundance of information to help patients with rare genetic diseases manage their daily living challenges. Amicus patient and professional advocacy are always looking for ways to educate people, by participating in relevant events and speaking on behalf of patients with rare diseases.


A rare disease called Fabry Disease leads to progressive, irreversible organ damage. This disease attacks the renal system as well as the nervous and cardiac system ( It also attacks various tissues. Amicus Therapeutics is in the process of developing a treatment for this disease. This is a devastating disease that attacks multiple organs. So far, this disease requires lifelong medical intervention.


Amicus Therapeutics is also striving to cure the Pompe Disease. Utilizing innovative technology, they are developing a topical therapy for Epidermolysis Bullosa, which is a rare disease causing blistering or erosion of the skin. This disease disfigures the patient and can be fatal. Currently, there is no approved treatment but the standard care consists of pain management and keeping the wounds clean and free of infections. Rare diseases matter to Amicus and they put forth great efforts to understand and find ways to cure these devastating diseases.