Here’s A Quick Guide To Understanding Pompe Disease
First thing’s first: What is Pompe disease? Pompe disease is a rare, degenerative muscle disorder that can impact an individual’s ability to move and breathe. How does Pompe disease affect the body? Pompe disease is an inherited disorder caused by a change in the acid alpha-glucosidase (GAA) gene. The change of the gene leads to […]
New Enzyme Therapy OK’d for Pompe Disease
The FDA approved avalglucosidase alfa-ngpt (Nexviazyme) on Friday as a new enzyme replacement therapy for Pompe disease. Avalglucosidase alfa is an intravenous drug that targets the M6P receptor, and is indicated for late-onset disease for those ages 1 year and older. In patients with the disease, which is marked by dysfunction of the lysosomal enzyme […]