Did you know Amicus Therapeutics is a Company Treating Rare Orphan Diseases?

Amicus Therapeutics Inc. is a public U.S based pharmaceutical company located in Cranbury, New Jersey. The company trading as NASDAQ: FOLD, went public in the year 2007. Before then, Amicus Therapeutics got funded by various venture capital funds including New Enterprise Associates, Canaan Partners and Radius Ventures.

Founded in 2002, Amicus Therapeutics focuses on specific rare disorders. That is in line with Amicus’ passion for treating devastating rare and orphan diseases. The global biotechnology company has a unique set of advanced development technologies to deal with a variety of rare human genetic diseases. The rare and orphan genetic diseases are Fabry disease, Pompe disease, and Epidermolysis Bullosa (EB).

What’s the goal of Amicus Therapeutics?

“We are passionate about what we do.” Through its CEO, John F. Crowley, Amicus Therapeutics is committed to having the needs of patients suffering from rare and orphan diseases at heart. The team is determined to make a significant difference to these patients and their custodians. The science-driven and business led company ensures they deliver meaningful benefits to their patients. They are at the forefront of potentially addressing unmet needs for communities with rare and orphan diseases.

Rare and orphan diseases information

Amicus Therapeutics specializes in providing treatment for rare and orphan diseases which include Fabry disease, Pompe disease and Epidermolysis Bullosa (EB). Here’s more about these diseases.

Fabry disease

Fabry disease caused by absence or deficiency of alpha-Gal A enzyme leads to accumulation of GL-3 and lyso-Gb3 cells causing continuous and irreversible damage of organs involving the renal, cardiac and nervous systems. The disease shortens the lifespan of affected men and women. Normally, cardiovascular disease is the cause of their early death. Currently, approximately 5000 to 10000 people are affected by the disease.

Epidermolysis Bullosa (EB)

EB is a chronic disease that erodes the skin. Patients suffering from EB experience painful blisters and wounds caused by even the slightest skin tear. Approximately 30,000-40,000 people in leading countries like U.S and Germany have been diagnosed with EB.

Pompe disease

Deficiency of enzyme GAA causes Pompe (SeekingAlpha). Its signs and symptoms are weak muscles particularly in the heart and skeletal muscles. Glycogen cells accumulate around leading to the cause of Pompe. An average of 5,000 to 10,000 people worldwide are suffering from Pompe disease.


Amicus Therapeutics, a rare company, specializes in providing treatment to people suffering from rare and orphan diseases. These diseases include Pompe, Fabry disease and Epidermolysis Bullosa. Through its CEO Crowley, Amicus Therapeutics has a mission of meeting the needs of people affected by these diseases. The team is committed and is at the forefront of using its advanced technology to provide significant treatment.

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