WebJul 9, 2024 · William Canfield is a glycobiologist, chief scientific officer and founder of an Oklahoma Citybased biotechnology company, Novazyme, which was acquired by … WebJan 19, 2024 · In 2006, the FDA approved the first treatment for Pompe disease. It consists of regular IV infusions of a man-made enzyme called alglucosidase alfa. The drug does the job of an enzyme in your body ...
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WebAug 7, 2001 · The most advanced of Novazyme’s product candidates is NZ-1001, an enzyme replacement therapy for Pompe disease that is on track to enter clinical trials by the end … WebAug 4, 2024 · Pompe disease, also known as glycogen storage disease type II (GSD II) or acid maltase deficiency (AMD), is a genetic disorder caused by a deficiency of the acid alpha-glucosidase (GAA) enzyme, due to recessive mutations in the GAA gene, which leads to accumulation of lysosomal glycogen [], diffusely but primarily affecting the skeletal and … chipley restore
Pompe disease - the real story: Novazyme
WebJohn Francis Crowley (born April 7, 1967) is an American biotechnology executive and entrepreneur and the chairman and CEO of Amicus Therapeutics.He co-founded Novazyme Pharmaceuticals with William … WebDiagnose and manage Pompe disease early to help your patients stay ahead of their disease. Pompe disease is a progressive, genetic neuromuscular disease that can affect patients of all ages. 1-3 Pompe disease can cause … WebPompe disease is an inherited disorder caused by the buildup of a complex sugar called glycogen in the body's cells. The accumulation of glycogen in certain organs and tissues, especially muscles, impairs their ability to function normally. Researchers have described three types of Pompe disease, which differ in severity and the age at which ... grants for cic