Amicus Therapeutics is a worldwide biotechnology company that is on the vanguard of rare and orphan diseases and especially focusing on Lysosomal Storage Fabry disorders, Epidermolysis Bullosa as well as Pompe disease. The effective running of the company is attributed to its rich technological and medicinal advancements aimed at developing cures for the named diseases. The Cranbury, NJ based company has its world revolving around their patients as each effort is funneled towards making their lives better.
Since Amicus Therapeutics was founded in 2002, the company has employed about 500 employees. It is through their efforts that the company has steered to resounding success. One of the biggest milestones has been in development of the migalastat- a personalized medicine that treats patients with Fabry disease depending on their genetic diagnosis. Amicus Therapeutics is also in the last stage development of SD-101, a product that could treat Epidermolysis Bullosa as well as the Enzyme Replacement Therapy, which Chaperone Advanced Replacement Therapy is working on as therapy for Fabry disease, Lysosomal Storage Disorders, and Pompe disease (GoogleFinance). Amicus Therapeutics boast of their largest bio program, ATB200/AT2221, a Pompe disease ERT that is designed to be administered alongside a pharmacological chaperone.
Some of the patients who have used their services have hailed their passion, which has leveraged the technology used for their treatment. Mike Stanzione, who had for nine years been misdiagnosed with Polymyositis, had grown into painful weakness until he was accurately diagnosed with Pompe disease. He suffered difficulties in breathing because his skeletal muscle had been debilitated. In 2011, thanks to Amicus Therapeutics, Mike became the first person in history to receive a diaphragm pacing system. The treatment allowed him to breathe independently, which he had been unable to do for 4 years, and reunite with his family. Vicki and Adriana who had the development of their children slowed down by Fabry disease, Sam from Southampton, the UK who suffered Pompe disease and Marlou from Groningen, Netherlands whose most members of her family have been affected by Fabry disease among others are all beneficiaries of the innovative products offered by Amicus Therapeutics (https://seekingalpha.com/article/4073303-amicus-therapeutics-small-biotech-seems-big-mo).