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Enzyme Replacement Therapy for Pompe Disease

Last updated Aug. 12, 2022, by Marisa Wexler, MS

✅ Fact-checked by José Lopes, PhD

Enzyme therapy for Pompe disease aims to “replace” the needed GAA enzyme

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.

Pompe Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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