An orphan condition is one that affects less than 200,000 people in the whole world. In a world of 7.5 billion people, that number is barely significant. It is hard enough living with such a disease, but patients endure the experiences alone or with barely a handful of people to share it. Pharmaceutical companies will usually take advantage of such cases because of how desperate they are. This is not only unethical but also immoral.
Enter Stage Left, Amicus Therapeutics
Amicus Therapeutics is an American biotechnology company operating out of Cranbury, New Jersey. The company has been on the forefront in the fight against rare and orphan diseases. The company’s biggest asset is its bold and driven employees who express pride in their business. The employees of Amicus Therapeutics are passionate and are committed to providing a sliver of hope for patients with rare and orphan diseases. They are dedicated to coming up with high-quality therapies as well as supporting rare disease communities.
Business-Led and Science-Driven
Amicus Therapeutics is committed to constant innovation (ReleaseFact). The company endeavors to obsolete its technology. Migalstat is a therapy in the late stages of development for Fabry. Fabry disease results from the abnormal deposits of globotriaosylceramide(a type of fat) in the blood vessel walls. Therapy is personalized on the basis of the patient’s genetic diagnosis. SD-101 is also in the late stages of development. It will be targeted towards relief from Epidermolysis Bullosa (EB). This genetic connective tissue disorder causes the skin to break out in blisters (https://yourbeautycraze.com/amicus-therapeutics-introduces-galafold-fabry-disease-patients/). Amicus Therapeutics is merging its resources to develop Enzyme Replacement Therapy for Pompe disease and other such disorders.
Amicus Therapeutics does not believe in gaining financial favor at the expense of patients’ well-being. Therefore, the company strives to give fair prices for their drugs and making them widely accessible. The company employees do not just talk about putting the patient. First, they put themselves in the shoes of the patient and the patient’s family. This way they are able to take well-thought-out maneuvers. The amicus Therapeutics chief aim is to provide meaningful benefit to the rare diseases communities.