Amicus Therapeutics Approves New Drugs for Those with Rare Conditions

Amicus Therapeutics is an American biopharmaceutical company that is based in Cranbury, New Jersey. This company joined the NASDAQ and went public back in 2007, which followed changing their abbreviation to AMTX in 2016 (YahooFinance). This is a therapeutic company that focuses on more rare diseases and offering prescriptions to help cure these conditions collectively known as a series of lysosmal storage disorders. The company is largely responsible for creating Chaperone-Advanced Replacement Therapy (CHART) platform and they are now concentrating on designing enzyme replacement therapies (ERT)s to help patients with another alternative to treatments for their condition.


While Amicus Therapeutics has no manufacturing power of their own they rely on outsourcing to other companies to help produce their medications. The company, however, has expanded as it opened a second site in 2008 out in San Diego, California before receiving a $500K donation from the Michael J Foxx Foundation to support their latest research with their CHART and ERT treatments at the David Geffen School of Medicine which is connected with UCLA. The company also received a $210,300 grant from the Alzheimer’s Drug Discovery Foundation to help support their pre-clinical work that they were doing in conjunction with the Icahn School of Medicine at Mount Sinai to help aid the Alzheimer’s Research Center (


Ultimately, all this research and the drugs they have come up with fulfills Amicus Therapeutics quest to make useful differences in the lives of patients suffering from various rare conditions that are associated with the Lysosomal Storage Disorders Fabry disease and Pompe Disease. The goal is to help make a difference in the lives of the patients suffering from these conditions and their caregivers by providing alternative treatments that may lessen some of the symptoms these patients are experiencing. This is the way that the Amicus Therapeutics can give back to the communities in which we all live and give to help make differences in people’s lives.