Who or What Is Amicus Therapeutics, Anyways?
Medicine is more advanced in today’s modern era than ever before. In the past 115 years, the average life expectancy was between 30 and 40 years of age. Just recently as 2014, the average Japanese woman’s living expectancy was well over 80 years of age, with the world average settling over 70 long, fulfilling years on planet earth. Without the many technologies, healthcare professionals use, people would die from minor illnesses far more frequently than with them, not to mention live shorter, less happy lives.
Amicus Therapeutics is a researcher and developer of medicines designed to treat ailments that few if any, medical research organizations have attempted to remedy. Rather than pushing out prescription drugs with plenty of manufacturers, Amicus Therapeutics cares about developing therapies for uncommon, “orphan” diseases. The biopharmaceutical company’s particular interests are in lysosomal storage disorders, making up roughly 50 ultra-rare diseases, rooting themselves in inabilities to properly metabolize food, water, and nutrients, directly as the result of faulty lysosomal functions.
Another two of the most popular illnesses Amicus Therapeutics is working on treating, is Epidermolysis Bullosa and Pompe disease. The latter ailment, Pompe disease, recently underwent the first phase of a clinical trial in which drugs ATB200 and AT2221, when taken together, were safe for use in those suffering from the painful condition. That safety study is slated to undergo the second and final phase later this year.
Amicus Therapeutics has done great things in striving to treat people suffering from Epidermolysis Bullosa (Yahoo). Usually shortened to EB, the condition is highly painful and entirely inconvenient for it suffers, causing innumerable blisters on one’s dry, exterior dermal layers, as well as in mucosal membranes, including the throat, esophagus, and nasal cavity. Fortunately for the improvement of society at large, Amicus Therapeutics is currently conducting an open label extension study in testing the potential wonder drug SD-101, a cream applied to painful skin lesions (https://www.facebook.com/pages/Amicus-Therapeutics/157577600941970). The organization is also undergoing the third phase of studying efficacy and safety of SD-101 cream, the same drug being tested in the aforementioned clinical trial.
The agency is currently headed by John F. Crowley.