Amicus Therapeutics at the Forefront of Rare Diseases

Amicus Therapeutics is a biopharmaceutical company that focuses on rare and orphan diseases. This is centered around disorders collectively called lysosomal storage disorders. They have concentrated on development of ERT’s (enzyme replacement therapies). The company does not manufacture on its own.

Amicus has a development for many treatments of human genetic diseases. They are based out of New Jersey. Their largest product is migalastat, which is for Fabry disease and only given to those who have been given a genetic diagnosis. They also have SD-101, this could potentially be the first-to-market therapy for Epidermolysis Bullosa, a rare genetic connective tissue disorder.

Amicus is working on developing enzyme replacement therapy products for Fabry disease, Pompe disease, and other Lysosomal Storage Disorders. They were founded in 2002 with the hope that they could help those with rare and devastating diseases that had no hope. The needs of their patients are the center of their scientific research, commercial organization, and clinical programs. This is not only for their patients, but also for those caregivers who travel with the patients every step of the way.

They are a company that loves to help others in their offices and out. They also do plenty of volunteer work as well. They have worked with Habitat for Humanity to help develop affordable housing. These houses are created to help people to be able to afford a living and raise their families.

Amicus is at the forefront of all of those rare diseases and advocating for those lives who cannot advocate for themselves. They work hard so that the fight for life is continuous and there is hope for those who are fighting that fight. There is hope for those fighting the diseases and the hope comes from the company and is given to the patients and their caregivers alike.

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