The next time you have a headache and reach for the ibuprofen, it is natural to be thankful that you have it available as you start to feel relief. There are many diseases, however, which can’t be treated with just an over-the-counter medication. Rare and orphan diseases, those diseases which don’t receive as much attention, and, therefore, funding, to treat, are some of the most devastating diseases precisely because of their uncommon occurrences. Amicus Therapeutics is seeking to change the devastating effects of rare diseases, one-by-one. It is a company devoted entirely to finding cures for these diseases that often no one else is seeking. View the market summary of Amicus Therapeutics at marketwatch.com.
Some diseases Amicus Therapeutics is researching are Fabry disease, Pompe disease, and other Lysosomal Storage Disorders, as well as the rare genetic connective tissue disorder Epidermolysis Bullosa. One of the challenges in treating these diseases is that the treatment often has to be personalized to each patient depending on their genetic diagnosis. One of Amicus Therapeutics’ greatest assets, though, is its laser focus on those individual patients, and its empathy with the personal situations they are facing as a result of having these rare diseases.
Everyone from the chairman and CEO of Amicus Therapeutics, John Crowley, to those on the front lines of research and development embody this commitment to the individual to ensure their passion and visions are never lost in the day-to-day minutiae of their work. There are pictures of patients they are working to treat, along with their individual stories, hanging in Amicus Therapeutics offices around the world. They also try to make each decision as if they themselves were living with whatever disease they are trying to treat to ensure that any risks they take or treatments they prescribe are achievable for those actually dealing with them. While their work is difficult at times, it’s also incredibly meaningful, and the entire company works to embody the spirit of those bravely fighting against these often devastating diseases. Read the company profile at Crunchbase.