Large biopharmaceutical companies often seek to develop and test a drug that can be used to either treat or cure the highest number of patients as possible. Unfortunately, the rarest diseases in the world get far less attention in the form of research and clinical trials of potentially life-saving therapies and drugs because not nearly as many people are affected by these diseases. This is where companies like Amicus Therapeutics steps in and takes on a very specific type of disease head-on. The company is based in Cranbury, New Jersey but has international offices in Germany, Netherlands, France, Italy, Spain and the United Kingdom with their international headquarters located in Buckinghamshire, United Kingdom.
Amicus Therapeutics is a biopharmaceutical company that specializes in what is known as enzyme replacement therapies. When some children are born their bodies do not produce critical enzymes within their bodies to speed up almost all of the chemical reactions that occur in a normal healthy child. If these reactions fail to take place or take place too slowly, the child can become extremely ill. So, Amicus Therapeutics attempts treat these diseases with their patented enzyme replacement therapies. Children in need are able to receive proper enzyme levels which in turn regulate the inner workings of their bodies. Amicus Therapeutics has received grants from many notable foundations such as The Michael J. Fox Foundation to support research being conducted at UCLA. This company has been working diligently since 2002 to attempt to eradicate a great number of the rarest diseases on the face of the planet. With their current leadership team and a team of scientists, they believe anything is possible (Glassdoor). Companies like Amicus Therapeutics make sure that no child’s disease is overlooked no matter how rare and is deserving of the same medical research and therapies available to all.