People today often wonder how to get proper medical care for some of the emerging orphan diseases. These diseases are often devastating and may have an expensive curative drug in the market due to their rarity. Such conditions include the Lysosomal Storage Fabry disease, the genetic skin disorder called Epidermolysis Bullosa and the Pompe Disease. Therapies for such diseases are uncommon, and one example of a company offering treatments to such diseases is the Amicus Therapeutics located in New Jersey U.S.A. Amicus Therapeutics is a biopharmaceutical company in New Jersey which offers therapies to treat rare and orphan diseases (Diseases which are rare and whose drugs to cure are often not in the market but mostly provided by the government).
Amicus Therapeutics, founded in 2002 became publicly known in the year 2007 under NASDAQ, a stock exchange market. The organization particularly deals with Lysosomal Storage Disorders. It majorly develops its products using Chaperone-Advanced Replacement Therapy and is currently aiming at developing enzyme replacement therapy products for Fabry Disease, Lysosomal Storage Disorders, and Pompe disease. Amicus Therapeutics has over 200 employees who work diligently to provide for patients’ needs. Amicus Therapeutics has its leadership layout from its CEO John Crowley, COO Bradley Campbell, and CFO William Baird.
The company shares its biggest accomplishments in the production of the migalastat, a medicine for the treatment of individuals with Fabry in the last stage. The drug, under the trade name Galafold, was developed and got its market in the year 2014, the company having no other product in the market until that time. Although having a large production of its products, the company does not have a manufacturing capability of its own and mostly relies on contract manufacturing (http://weeklyopinion.com/2017/08/amicus-therapeutics-announces-the-launch-of-new-drug/). Amicus Pharmaceuticals has also had more achievements in the past years has expanded its site from a single one in New Jersey and set another location in San Diego. The company has also received several grants including US$500,000 grant from The Michael J. Fox Foundation and another US$210,300 grant from Alzheimer’s Drug Discovery Foundation to support its work. The company plans to develop a biologics program called ATB200/AT2221 which is a Pompe Disease ERT (enzyme replacement therapy) (DialDish).