Thousands of rare diseases afflict humans. Rare diseases, commonly known as orphan diseases are serious health conditions that can be life-threatening. A significant number of these diseases lack appropriate treatments and medications due to inadequate investment in research and drug development by the relevant organizations.
According to a report released by the National Institute of Health, rare diseases affect about 200,000 people all over the world. These numbers might appear small, but they cause serious trauma, especially to the affected people and those close to them. The figures also indicate that there are about 7,000 rare diseases, with a huge portion of them being extremely rare.
Development of drugs for rare diseases is an utterly expensive endeavor that needs for substantial resources to be invested in research. The amount of funds being spent in research for rare conditions has been unsatisfactory. This is mostly because of chronic diseases such as cancer and diabetes; rare diseases occur at extremely low frequencies. Besides, it takes a lot of time to conduct research and clinical trials before a drug for treating rare conditions is released to the market. It can take up to twelve years to create a drug for human use. Read more about Amicus Therapeutics at seekingalpha.com.
Involvement of Amicus Therapeutics
Amicus Therapeutics is one of the few companies that has taken the unbeaten path to revolutionize treatment for rare diseases. It is a biotechnology-based company that specializes in the research and development of drugs for various rare diseases. The company is listed under the Nasdaq as Fold.
Amicus Therapeutics has devoted a lot of their time and resources to find cures and treatment for the world’s most rare diseases. One of the most renowned treatments developed by Amicus Therapeutics is migalastat.
Migalastat is a special medication created for the treatment of a rare disease known as Fabry. FDA recently passed Migalastat and it is now available in the market in Europe, Israel, and Switzerland. Amicus Therapeutics is pushing for the drug’s approval in the U.S., Australia, Canada, and Japan. The company also recently introduced SD-101, a one-of-a-kind medication used in the treatment of Epidermolysus Bullosa. The company is conducting research on therapies for a variety of rare conditions such as Pompe and other lysosomal disorders. Visit Google Finance to know more about Amicus Therapeutics.