Amicus Therapeutics, research leader in rare and orphan diseases.
Amicus Therapeutics is new Jersey-based biopharmaceutical company whose main focus is the development of technologies used in therapy for treatment of a wide range of diseases focusing more on rare and orphan diseases. In fact, the emphasis has been given to three key elements as the basis for the development of therapeutic remedies. The disease has to be rare, the technology must have potential to be top-notch quality and data available or collected must translate to significant benefits for patients.
The company has also taken upon itself to cultivate personal and compassionate approaches to patients as well as their caregivers and the entire family at large. This culture has been heavily underlined in the company’s focus especially in the advancement of treatments for CDKL5 deficiency Lysosomal Storage Disorders as well as Epidermolysis Bullosa.
Amicus has set a patient advocacy forum to know as Amicus Patient & Professional Advocacy which partners with various stakeholders such as healthcare practitioners, patients, caregivers and families and other concerned parties to deliver the highest quality support system for persons living with various rare conditions.
The company has its international headquarters in Buckinghamshire, United Kingdom while operating from other countries including Italy, France, Spain, Germany and the Netherlands. Its corporate headquarters is in Cranbury, New Jersey, USA.
The team of dedicated professionals at Amicus Therapeutics, is led by Chairman John F. Crowley. His background and relationship with therapeutic cure go back to whose background goes way back to1998 when two of his children were diagnosed with a rare and severe neuromuscular disorder known as Pompe Disease. He co-founded Novazyme Pharmaceuticals which immersed itself in research on the Pompe Disease and was of significant contribution to saving the children’s lives. The company was later sold out but Crowley remained Senior Vice President. Amicus is thus in the hands of a man who understands the effects of rare diseases.
Amicus Therapeutics has partnered with several organizations over time and recently applied a request for authorization to market migalastat a drug meant to cure Fabry disease.