Amicus Therapeutics: Dedicated to Providing the Best Solutions for Rare Diseases

Amicus Therapeutics is a biotechnology company dedicated to developing advanced therapies for rare diseases. The firm was founded in 2002 and went public in 2007 under its trading symbol FOLD, which is under the NASDAQ stock market. Amicus Therapeutics has made a significant impact in their area of specialization and has achieved global recognition for their commitment to offering solutions to people living with the rare disease. In 2014, it was recognized as holding the largest collection of small molecule pharmacological chaperones in the pharmaceutical industry globally. Through intensive research, the company develops medicine technologies and drugs to assist people with rare diseases such as Fabry diseases, Epidermolysis Bullosa and Pompe disease. Amicus Therapeutics is a global leader in the treatment of most genetic disorders. It is based in Cranbury, New Jersey and has a research unit in San Diego.


Recently, FDA gave Amicus Therapeutics the go-ahead to submit an NDA (New Drug Application) for migalastat, an oral precision drug for Fabry disease. The CEO and chairman of Amicus Therapeutics, John F. Crowley, and Chief Medical Officer, Jay Barth, expressed confidence that FDA will approve the drug because the clinical data that supports this medicine is science based and will help many lives (YahooNews). Amicus Therapeutics also has another product known as SD-101, a possible treatment for the connective genetic skin disorder, Epidermolysis Bullosa that is also in its last development stage. Additionally, they have a program known as ATB200/AT2221 for the Pompe disease therapy.


Amicus Therapeutics is very supportive of the rare disease community (,-NJ-jobs.html). Through the Amicus Patient and Professional Advocacy platform, the company works in partnership with patients, caregivers, care practitioners, and institutions to offer support to the rare disease community through different initiatives. These include holding educative forums on the disease, consulting patients before developing treatments and diligently seeking better therapies.


What makes Amicus Therapeutics such a huge success is that it is composed of a dedicated team who are very passionate about offering the best solutions to the patients. They uphold integrity in their operations and use technology to deliver the appropriate treatment for human genetic disorders by conducting thorough scientific research.