With sites in Cranbury, New Jersey, San Diego, California, and locations in Europe, biopharmaceutical company Amicus Therapeutics is at the forefront of research and treatment for lysosomal storage disorders. These are typically referred to as rare and orphan diseases and include Fabry disease, Pompe disease, and Epidermolysis Bullosa. Amicus Therapeutics has put their their emphasis on enzyme replacement therapies (ERTs)for these disorders. They currently have in development the drug migalastat, an oral pharmacological chaperone that will be used in the treatment of Fabry Disease. Patients with Fabry disease can experience a wide range of symptoms that can include kidney complications, heart disease, and an increased risk of stroke. Chaperone-Advanced Replacement Theraphy is being developed to treat Pompe Disease, a metabolic disorder which can severely damage nerve and muscle cells in the body. A topical therapy known as SD-101 is in development for the treatment of Epidermolysis Bullosa, a rare genetic connective disorder. Patients with EB experience painful tears and skin blisters, often resulting from only slight friction.
Amicus Therapeutics is serious about patient advocacy and works to support medical professionals, caregivers, patients, and patient organizations. They seek feedback from the families and patients that have lysosomal disorders in order to give them the best possible information for dealing with their conditions on a regular basis.
Amicus Therapeutics has received grants from the Alzheimer’s Drug Discovery Foundation and The Michael J. Fox Foundation (SeekinAlpha). They support programs nationwide to help improve patient care and to provide the best information available to healthcare providers. On February first of this year, Amicus Therapeutics sponsored Rare Disease Day in order to bring attention and support to those who struggle with rare conditions, with events being held
in the United States and Europe.
At present, the Chief Executive Officer of Amicus Therapeutics is John Francis Crowley and the President is Bradley L. Campbell. The company uses contracted manufacturing to develop their products.
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