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Sep 22, 2023 · How is Pompe disease treated? Pompe disease treatment includes enzyme replacement therapy (ERT). Your healthcare provider will give you one of the following medications intravenously (through your vein): Alglucosidase alfa. Avalglucosidase alfa. These drugs are genetically engineered enzymes that act like naturally occurring enzymes. They help:
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May 6, 2022 · The treatment of Pompe disease is disease-specific, symptomatic, and supportive. Enzyme replacement therapy (ERT) is an approved treatment for all people with Pompe disease.
Jan 19, 2022 · 5 min read. Doctors can’t cure Pompe disease. But treatment can help babies, children, and adults with the condition live longer, and often with fewer complications. In 2006, the FDA...
Pompe disease (aka glycogen storage disease type II [GSD II] or acid maltase deficiency [AMD]) is a rare autosomal recessive genetic disorder1,2. Epidemiology. • Globally, prevalence of ~1 in every 40,000 people1. • Three-quarters of these are late onset and one-quarter are infantile1.
Current approaches to all types of Pompe disease are tailored to specific patients and may include enzyme replacement therapy (ERT), and additional supportive therapies that target specific symptoms and complications of the disease.
Jun 10, 2022 · 4 min read. What Is Pompe Disease? If you or your child has Pompe disease, it's important to know that each case is different, and that doctors have treatments to help manage it. Pompe disease...
Jul 19, 2024 · Alglucosidase alfa (Myozyme©) to treat infantile-onset Pompe disease. Lumizyme© to treat individuals of all ages with Pompe disease. Avalglucosidare alfa-ngpt (Nexviazyme©) for individuals age 1 and older with late-onset Pompe disease. Learn About Clinical Trials.
