Amicus Seeks Cures For Many Orphan and Rare Diseases

Amicus Therapeutics, created to find a cure for orphan diseases, was founded in Cranbury NJ in 2002. After five years of private research, Amicus Therapeutics went public. In 2008, the therapeutics company opened a branch in San Diego, California. Amicus presently has locations in Germany, Spain, France, the Netherlands, the United Kingdom, and Italy. The research company relies on many donations from fundraising organizations that include a $210,300 grant from the Alzheimer’s Drug Discovery Foundation and a $500,000 grant from the Michael J Fox Foundation, known for treating Parkinson’s.

Recently, Amicus Therapeutics (:Small Biotech Seems To Have The ‘Big Mo’) is working on treatment for Fabry disease. Fabry disease is damage to the peripheral nerve fibers that can effect the GI tract, including the kidneys and the heart. It can be diagnosed early in childhood. Fabry disease is considered a lysomage storage disorder. Before Amicus Therapeutics came along, Fabry disease was a fatal disease. Marlou from the Netherlands suffers from Fabry disease that took the life of her grandmother. Marlou’s sister, mother, and aunt currently suffer from Fabry disease. In the Netherlands, it was wrongly assumed that women can only carry Fabry disease and not get it. Marlou knows this isn’t true. A sufferer herself, she isn’t going to let this disease stop her from enjoying life. She’s being treated with Migalistat.

Amicus is working with genetic diseases by researching how to target mutated proteins. In 2007, Mike from Saddlebrook, New Jersey, had a breathing problem that was misdiagnosed. Finally, in 2016 doctors learned he had Pompe disease that is a form of muscular dystrophy. MD has similar symptoms as Polymiositis, also a muscular disease. If Mike hadn’t been diagnosed with Pompe disease and received treatment, he might have died.

Amicus Therapeutics has a patient advocacy program that not only treats these rare, orphan diseases, it also educates the patient to help in treatment. In fact, Amicus is at the forefront of treating orphan diseases. Amicus is the first pharmeceutical company to market therapy for Epidermolysis, a muscular connective tissue disease. The pharmeceutical company is working on chaperone-Advanced Replacement therapy for the many of the orphan and rare diseases.

 
Need more information on Amicus Therapeutics? Visit: https://www.glassdoor.com/Reviews/Amicus-Therapeutics-Reviews-E26068.htm

Leave a Reply

Your email address will not be published. Required fields are marked *