Rare and orphan diseases can sometimes be a particularly disheartening segment of medical research because of the limited amount of organizations and companies looking into such things. However, it is important to stay positive about those who are blessed enough to make efforts in that direction, and Amicus Therapeutics in one such company that has encouraging results. Lysosomal storage diseases have been a particularly promising niche in that regard as well (WeeklyOpinion).
There are three primary programs which Amicus Therapeutics focuses on in order to make a difference in patient lives (https://www.google.com/finance?cid=706165). There is the Fabry Disease Program, Epidermolysis Program, and the Pompe Disease Program. Through these and some other preclinical programs, the company has a development pipeline which is quite promising with things like Migalastat (trade name Galafold) and other enzyme replacement therapies (ERTs) being continuously worked on. Amicus Therapeutics has had some setbacks as could often be expected in any such a program that is at complicated as theirs, and they have been fortunate to receive help from such organizations as the Michael J. Fox Foundation so that they can continue to stay in positive territory.
Amicus has acquired competitor Callidus Biopharma and Scioderm while also working in cooperation with the Alzheimer’s Drug Discovery Foundation to push the envelope further in that direction as well. The company continues to be a strong buy for those hoping to invest in stocks with a potential to return well. They are continuing to work with the FDA in order to get Migalastat (trade name Galafold) to market, and if and when they are finally able to release that to consumers with confidence that it does what they are saying it does then the holding will probably soar high. The ticker on the NASDAQ is FOLD, and there are many other reasons to check out this buy if the money is there to make the investment. The company is doing important work that should leave the planet a better place when they are able to fully release these type of treatment programs to the public increasing the overall health of rare and orphan disease carriers.