Amicus Therapeutics is based out of Cranbury, New Jersey. As a biotechnology firm, Amicus Therapeutics strives to research genetically based diseases. Many people have rare diseases that they know nothing about (Philly Purge). Amicus Therapeutics strives to serve this community. The main focus, however, is on lysosomal storage disorders. The company chooses to work on enzyme replacement therapies as well as lysosomal storage disorders. This company believes in passion for what they do and looks for driven personalities for staff. They like pushing ideas as far as possible, as well as asking tough questions. This company upholds high standards of integrity in addition to taking full responsibility for their actions. Part of the companies’ philosophy is to sponsor teamwork. Amicus Therapeutics also likes to emphasize work-life balance in addition to communicating openly with honesty as well as respect. What a lysosomal storage disorder actually come from a group of 50 rare metabolic disorders. Lysosomes are enzymes in cells, which process large molecules. Sometimes these enzymes do not work right causing a mutation. Cells die. Lysosomes are basically able to recycle material into more wanted substances. All genetic diseases are based on DNA inheritance patterns from both parents.
Children with these illnesses die within a few months of birth. These disorders are characterized by the type of defective protein. Amicus Therapeutics strives to develop a new treatment for affected individuals (https://finance.yahoo.com/quote/FOLD?ltr=1). Amicus Therapeutics works on the following illnesses, Fabray disease, Epidermolysis Bullosa, Pompe Disease, and CDKL5. Fabray causes organ failure, epidermolysis bullosa causes blisters on the skin. Pompe disease is about muscle weakness in the body, and CDKL5 impairs neurological development. Having a rare genetic disease is challenging, the company strives to create a means of patient support. Patient input is important to them because it helps with clinical trials. This companies’ main goal is to strive for better treatments.