Amicus Therapeutics Helps Find Treatments for Rare Orphan Diseases

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Amicus Therapeutics is a biopharmaceutical company based in Cranbury, New Jersey. The company’s focus is on finding advanced therapies and treatments for a broad range of human genetic diseases. These diseases, known as orphan diseases, typically affect fewer than 200,000 people nationwide (Google Finance). Currently, the company’s main concentration is on treatments for Lysosomal Storage Disorders such as Fabry disease and Pompe disease.

 

Amicus Therapeutics has directed their development on the Chaperone-Advanced Replacement Therapy (CHART) and enzyme replacement therapies (ERT). This kind of medical therapy replaces an enzyme that is either absent or deficient in the body. The patient is typically administered an IV, which contains a solution of the missing enzyme. ERT has been effective at helping patients with lysosomal storage disorders.

 

In order for the company to invest in a program, it has to meet three specific criteria. It needs to be considered a rare disease, the technology must have the potential for being at the top of its class, and the clinical data must provide patients with significant benefits (https://yourbeautycraze.com/amicus-therapeutics-introduces-galafold-fabry-disease-patients/). Amicus Therapeutics is dedicated to finding treatments for these rare and devastating diseases. Currently, the company’s most advanced product candidate is migalastat. Migalastat is a pharmacological chaperone treatment for Fabry disease. Other product candidates include SD-101, a treatment therapy for Epidermolysis Bullosa, and ATB200/AT2221, an ERT for Pompe disease.

 

John F. Crowley has been the CEO of Amicus Therapeutics since 2005. Mr. Crowley’s dedication to the field stems from the diagnosis of two of his children with a severe neuromuscular disorder known as Pompe disease. Under Crowley’s leadership, the employees constructed a belief statement that summarizes the company’s core values and principles. It states: “We believe in the fight. We believe in our future. We believe in each other.” It is evident that Amicus Therapeutics is truly driven by the desire to find medical treatments for patients and families suffering from these rare and debilitating diseases.