Amicus Therapeutics is a biopharmaceutical firm that is based in Cranbury New Jersey. The company focuses on rare as well as orphan diseases, collectively referred to as Lysosomal Storage Disorders. The product the company develops is solely based on Advanced Replacement Therapy. It has laid emphasis on the development of the enzyme replacement therapies, commonly referred to as ERTs. The year 2014 will go down in history as the year that Amicus came up with the most elaborate portfolio of the small molecule pharmacological chaperones in the vast pharmaceutical sector. The company is very passionate about what they do and have embraced constant innovation.
The company is headed by the able leadership of John F. Crowley, who is the chairman as well as the chief executive officer. He has been able to accumulate a lot of knowledge in the field of biotechnology from the year 1998. This is the year when his two children were diagnosed with the pompe diseases (DialDish). This necessitated John to find treatment for them and he indulged in intensive research trying to find the cure for pompe disease. This was how he was able to save his children.
Amicus has no manufacturing capabilities and, thus, relies on others’ companies by signing manufacturing contracts (https://www.glassdoor.com/Reviews/Amicus-Therapeutics-Reviews-E26068.htm). Amicus Therapeutics believes in delivering meaningful benefits to the clients. The company strives to provide unrivaled therapies in a bid to solve the rare as well as orphan diseases that may prove to be devastating.
Amicus Therapeutics is in the developmental stage of coming up with oral pharmacological chaperone migalastat that they believe will be a breakthrough as far as the treatment of Fabry disease is concerned. Fabry is a disease that is caused by the mutation in GLA gene. This disease results in irreversible organ damage. The symptoms of Fabry disease may be different from one patient to the other. Migalastat is the company’s lead product. Other programs that Amicus Therapeutics is undertaking include Epidermolysis Bullosa (EB), which is characterized by blisters on the skin as well as lesions. The company has been very instrumental in trying to manage and treat various diseases.