Amicus Therapeutics is a pharmaceutical company that develops medications, and drug treatment plans for people with rare genetic disorders. The company was first established in Cranbury, New Jersey in the year 2002. Amicus Therapeutics has grown tremendously since then and became a publicly traded company in 2007. Amicus Therapeutics specializes in developing drug treatments for people with lysosomal storage disorders. Lysosomal storage disorders are a category of genetic disorders that affect the body’s ability to metabolize fats and proteins.
People who were born with a lysosomal storage disorder need constant medical treatment in order to live a normal life. If left untreated, a person with a lysosomal storage disorder will suffer from nerve damage, organ failure, and eventually, would die.
Amicus Therapeutics will change the lives of thousands of people through the development of new drugs, like migalastat.
Migalastat is currently the only medication made by Amicus Therapeutics that is out on the market. Migalastat is only available in Europe, where they have branded it as Galafold. Galafold is used in the treatment of Fabry Disease. There is no cure for Fabry Disease.
Although Amicus Therapeutics is not the first company to develop this kind of medicine, it enters a section of the pharmaceutical industry where there is a lot of room for growth (YahooFinance). People with these kinds of rare diseases will need as many options as they can get.
People with Fabry Disease suffer from:
- Gastrointestinal problems
- Burning sensations in the hands and feet
- Hearing loss
- Cloudy vision
- Joint pain
- Abdominal pain
On July 11, 2017, Amicus Therapeutics got permission from the U.S. FDA to submit a new drug application for migalastat. There is already a lot of data from clinical research trials which support the effectiveness of migalastat. Migalastat works by replacing the patient’s defective enzymes with stable ones, and when this happens cells can begin removing waste normally.
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