Registered as a biopharmaceutical company, the company is set to make a difference on lysosomal storage disorders. This disorder is a collective term used to refer to roughly 50 metabolic disorders that are life threatening. Anyone who has this disorder is expected to have development delays. These patients may have pulmonary and cardiac problems, enlarged livers and spleens and may even have bones that grow at very abnormal rates. These patients may also suffer from dementia, seizures, deafness and even blindness.
As of 2014, the business doesn’t have any marketed therapeutics. It continues its quest in finding the right medication to treat rare diseases related to lysosomal storage diseases (LSD). Through its partnership with other companies, Amicus Therapeutics is able to further advance its studies on the products they are developing. They already have several products formulated based on their Chaperone-Advanced Replacement Therapy (CHART) and they are pursuing the advancement of these medications. These breakthroughs are in various stages of clinical trials and are nearing their approval stage.
Amicus Therapeutics has taken on the challenge of finding the cure for these rare diseases. Through the funds the company has received from investment firms, they continue to provide hope to those patients diagnosed with these rare diseases. Using the advanced technology brought by small molecules, the business is set to take the challenge posted by Fabry disease and Parkinson’s head on. Amicus is headquartered in Cranbury, New Jersey and trades publicly on the NASDAQ, using the symbol FOLD.