Amicus Therapeutics: Developing Treatment for Rare and Orphan Diseases

Rare and Orphan Diseases

The world is home to all kinds of diseases. There are the common diseases that affect a large population in the world, and there are also the orphan and rare diseases that affect a small number of people. A good example of orphan diseases is the Lou Gehrig’s disease, Cystic Fibrosis, and Tourette’s syndrome. It is important to note that, orphan diseases fall under rare diseases. However, an orphan disease is so rare that it lacks the support for research and discovering treatments for it. But in every situation, there is a silver lining, and in this case, the silver lining comes in the form of a biopharmaceutical company known as Amicus Therapeutics.


About Amicus Therapeutics

Founded in 2002, Amicus Therapeutics is a biotechnology organization that focuses on infrequent and orphan diseases. The headquarters of the firm are in Cranbury, New Jersey. The company has been under the NASDAQ trading symbol FOLD since 2007. It is using modern technology to come up with possible treatments for these rare and orphan diseases. They have a particular interest in Lysosomal storage disorders.


Product Development

Amicus Therapeutics has made great strides in their product development. One of their leading medical products is the migalastat medicine ( This medicine was developed to treat individuals who suffer from Fabry disease. They are also developing SD-101. This product is being developed to treat Epidermolysis Bullosa. It is a genetic connective tissue disorder. Amicus Therapeutics heavily relies on the Chaperone-Advanced Replacement Therapy platform. In 2010 to 2013, the company partnered with GlaxoSmithKline and JCR Pharmaceutical to study co-formulation with recombinant alpha-galactosidase.


This company wants to make a difference in the society (ReleaseFact). The fight to treat and eradicate rare and orphan diseases is their core purpose. Fortunately, they are being championed by organizations that show their support through the funding of their projects. They have received funding from organizations such as the Michael J. Fox Foundation and Alzheimer’s Drug Discovery Foundation. People suffering from rare and orphan diseases have a reason to smile. With companies such as Amicus Therapeutics in the forefront, there is hope for the afflicted.