Category Archives: Biopharmaceutical

Innovative Approach To Treatment of Rare Diseases

Exciting new innovations in medicine have opened up new treatment options for those who suffer from rare and orphan diseases. Amicus Therapeutics is a biopharmaceutical company that is pushing the boundaries in terms of treatment approaches for rare diseases. They are focused on a group of diseases called Lysosomal Storage Disorders.


The treatment options they are researching and developing include chaperone-advanced replacement therapy and enzyme replacement therapy (YourBeautyCraze). The researchers and scientists of Amicus Therapeutics have several treatment methods in focus. One of these includes a drug called migalistat, which is currently in late-stage development. It is designed to treat people with Fabry disease.


All of the work that Amicus Therapeutics does is aimed at changing the lives of those they treat. The rare and orphan diseases that this company focuses on affect people across the globe and these people have had to struggle with diseases that are rare and debilitating.


The treatment options for these diseases have been limited, and Amicus Therapeutics aims to change that with cutting edge biomedical innovations. The philosophy of the company is to not be limited by prior beliefs and thoughts about what is possible. They continuously push the boundaries and try new techniques. This philosophy has allowed them to be leaders in the treatment of rare and orphan diseases world wide.


Amicus Therapeutics has headquarters in New Jersey, USA, and also has a branch in California, USA (Google Finance). They do not manufacture their own drugs but instead work on a contract basis with off site manufactures. The resources of this biopharmaceutical company are focused on research, innovation, design, and working with patients and families.


Over the past ten years, this innovative company has received recognition from prominent funds such as the Micheal J. Fox Foundation and the Alzheimers Drug Discovery Foundation. This support has gone towards research efforts in partnership with various medical schools.


In addition to this funding, the company has grown due to acquisitions of other groups that were also studying rare diseases. For example, the acquired a company that had been studying enzyme replacement therapy for treating Pompe disease. This acquisition, in turn, boosted Amicus Therapeutics own knowledge regarding Pompe diseases and allowed them to better help their clients who suffer from this ailment (