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 […]