Amicus Therapeutics, a biopharmaceutical company based in New Jersey, is a leader in the fight against a fatal group of genetically inherited diseases called Lysosomal Storage Disorders. A main component of the fight that this innovative company has launched is called enzyme replacement therapy.
What Are Lysosomal Storage Disorders?
These are a rare group of disorders, also referred to as LSDs, are fatal malfunctions of the body that affect the lysosomal organelles of the cell. The lysosomes functions within human cells as a “cleaning unit” that helps the cell get rid of toxic wastes, among other things. People with LSDs have lysosomes that are not functioning properly and therefore disrupting the way the body processes food and makes energy.
What Is Enzyme Replacement Therapy?
Enzyme Replacement Therapy, or ERT, is a therapeutic intervention that is based on replacing the enzymes that are missing or deficient in a patients body. Usually, this is done through an Intravenous route. Enzymes are important chemicals that help the human body complete the processes of living.
What Are Some Examples of Diseases That Are Treated With ERT?
Though ERT was first developed in 1964, it is still being actively studied as far as the ways it can be applied in real life. Amicus Therapeutics has been working hard towards getting ERT into the lives of the people that need it. Amicus Technologies takes great care in working with patients and their families towards implementing treatments. Currently, the company is working on completely new enzyme replacement therapies for Fabry diseases.
What Is Amicus Therapeutics?
Amicus Therapeutics is a company that strives to develop treatments for rare and orphan diseases. They have worked specifically with patients who have rare diseases such as Fabry disease, Epidermolysis Bullosa, and Pompe disease. This company works closely with the technology associated with ERT, as well as another technique called CHART (which stands for chaperone replacement therapy). Both approaches have led the company to pioneer treatment options for those who suffer from rare diseases. The company has been supported by foundations that help in the search for cures. They are based in New Jersey and have a work site in California as well.