Amicus Therapeutics: Managing Orphan and Rare Diseases in the Society

There are 39 categories of diseases in the world. Out of these diseases, we have the rare and orphan diseases. Orphan and rare disease are generally diseases that affect less than 200,000 people in every country. Companies like Amicus Therapeutics, specialize in advanced therapies to treat rare and orphan diseases. Amicus Therapeutics was founded in 2002 but went public in 2007(MarketWatch). The company has 201 to 500 employees, with its headquarters being in Cranbury, New Jersey.



The company uses Chaperone-Advanced Replacement Therapy in developing its products (GoogleNews). They are the producers of Migalastat, a medicine that treats people with Fabry disease. This company generally concentrates on enzyme replacement therapies development. The success of Amicus Therapeutics has placed it at the forefront of the pharmaceutical industry. It has received recognition in having the biggest portfolio of small molecule pharmacological chaperones.


The orphan diseases Amicus Therapeutics deals with include; Genetic Connective Skin Disorder Epidermolysis Bullosa, Lysosomal Storage Disorders Fabry Disease, and Pompe Disease. Amicus Therapeutics seeks to help the lives of afflicted individuals by providing them with accessible medical care. The company takes great pride in their biologics program ATB200/AT2221.


Patients’ Reviews

A lot of people have benefited from the work Amicus Therapeutics is doing. Many of the patients treated for these orphan diseases have testified to how their health improved after embarking on the orphan medicine created by Amicus. The company is able to touch the lives of these people not only through their medicine but also through clinical programs and commercial organization.


The company continues to grow under the management of its CEO John Crowley. Crowley can empathize with a lot of these patients because he has a daughter who suffers from Pompe disease. The company has also expanded by launching Galafold in the European Union. Amicus Therapeutics has also been listed in the ten best companies where investors can buy their stocks.


Amicus Therapeutics has helped save the lives of people with rare diseases, which are barely recognized in the society. Through their continued scientific innovation, they continue to give individuals around the world some hope.

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