August 22, 2014
Amicus Therapeutics (NASDAQ: FOLD) reported this week positive data from study 012 of its chaperone small molecule migalastat (Amigal) in patients with Fabry disease. Fabry is a rare disease that affects about 1 person in 117,000 people or about 5,000 patients evenly distributed between Europe and the United States. The hallmark of the disease is the accumulation of the substrate globotriaosylceramide (Gb3) in the kidneys. For more information on Fabry, visit this link.