Amicus Therapeutics- A Leader in Rare Disease Research and Development

Amicus Therapeutics is a biopharmaceutical company based out of New Jersey. Amicus Therapeutics has a focus on rare diseases with a specific focus on orphan diseases categorized as lysosomal storage disorders. The company has worked diligently on developing products based on the Chaperone-Advanced Replacement Therapy platform also known as the CHART® platform.

The company which was founded in 2002 went public in 2007 on the NASDAQ market under the trading symbol FOLD.The companies leading product candidate which is still in late-stage development goes by the name of migalastat and is designed in hopes of treating Fabry disease. Another product that is also in the late stages of development is known as SD-101. This product was developed in hopes of treating a disorder known as Epidermolysis Bullosa (EB) which is a rare genetic connective tissue disorder. Learn more about job offerings of Amicus Therapeutics at Indeed.

Amicus Therapeutics may be based out of New Jersey however they have locations worldwide. You can find Amicus Therapeutics in the United Kingdon, Germany, France, Italy, the Netherlands, Spain, and Japan. To give you an understanding regarding the types of conditions Amicus Therapeutics is hoping to address, check out the information below.

Fabry Disease is a genetic mutation of the GLA gene. This disease causes irreversible damage to vital organs and tissues throughout the body and requires lifelong medical intervention to manage the damage it does to almost every organ system in the body. The disease is currently managed by Enzyme Replacement Therapy but has no cure. Amicus Therapeutics is hoping to change this with their product migalastat.

Epidermolysis Bullosa also known as EB is a rare genetic disorder that causes erosion and/or blistering of the skin as well as the epithelial lining of other organs in certain cases. This disorder is potentially disfiguring and can sometimes be fatal. Currently, there are no approved treatments for EB. Patients current treatments consist of pain management and infection prevention. Hopefully, this will change if the product by Amicus by the name of SD-101 is approved. Visit to know more about Amicus Therapeutics.

When it comes to research and development to help patients suffering from rare and orphan diseases, Amicus Therapeutics is a leader.