Amicus Therapeutics: Paving the Way for Rare Disease Treatments

Amicus Therapeutics is an American biopharmaceutical company. They are currently based in Cranbury, New Jersey. The company’s primary focus is research and treatment of rare and orphan diseases.

Rare and orphan diseases are those which affect a small amount of the population. Because of the rarity, there are always fewer options for individuals to contract these conditions and some of them suffer from having mismanaged cases to having to treatment options at all.

Amicus Therapeutics aims to solve this problem by making more treatment options available for patients with rare and orphan diseases. At present, they are one of the pioneers of advanced therapies for treating rare conditions. They have developed a wide variety in treatment of genetics-related diseases. They are currently improving their growth as a global biotechnology company, with their goal of developing products that involve altering the causes that are based on human genetics.


In the forefront of their product developments is migalstat. It is described as a medicine that will help treat patients with Fabry disease. Fabry disease is a rare condition due to the lack of a specific enzyme. This disease affects several vital parts of the body including the kidneys, heart, and the whole nervous system. Aside from this, Amicus Therapeutics also developed a treatments for Epidermolysis Bullosa, Pompe Disease and other Lysosomal Storage Disorders. Their continuous research has given tremendous contributions to the improvement of rare disease treatment. Visit to know more about Amicus Therapeutics.

Amicus Therapeutics has come a long way since its launch in 2005. Their core values show their passion and boldness to provide healthcare options for individuals who otherwise have none. Amicus Therapeutics believes that it is essential to provide high-quality treatment for individuals living with rare diseases, show support in the whole community including the families of the affected, be passionate in what they do, and be flexible to embrace the innovations ushered by advancements in medicine.

As one of the few companies in the medical field of rare diseases, Amicus Therapeutics hold fast to their principles that the center of their science is for the needs of their patients. They hope to bring a positive different in the lives of the affected individuals and their families. Follow Amicus Therapeutics on Twitter.