FAQs about enzyme replacement therapy for Pompe disease
While other treatments may be administered to help manage the symptoms of Pompe disease, enzyme replacement therapies are the only approved treatment that directly addresses the root cause of the disorder in order to slow disease progression.
There are two enzyme replacement therapies approved in the U.S.: Lumizyme and Nexviazyme. Lumizyme is approved for all forms of Pompe disease, while Nexviazyme is approved for patients with late-onset disease, ages 1 and older.
Both approved enzyme replacement therapies, Lumizyme and Nexviazyme, are administered intravenously, or via an infusion into the bloodstream, every other week.
Enzyme replacement therapy is not a cure for Pompe disease, but it can slow the disease’s progression.