Newly Diagnosed: Treatment Strategies for Pompe Disease

While Pompe disease does not yet have a cure, there are therapeutic approaches that can help manage the disease, easing symptoms and improving quality of life. Several experimental treatments also are currently in clinical trials that could prove beneficial for Pompe patients. Check out the information below to explore more about therapeutic strategies your doctor may recommend and what therapies are in the pipeline for potential future use.

Approved Treatments

Enzyme replacement therapy is the current standard treatment for patients with Pompe disease. This approach is used to increase the levels of GAA in the body and reduce the accumulation of glycogen inside cells. Lumizyme (alglucosidase alfa) is the only approved ERT for Pompe in the U.S. It is available in the EU under the brand name Myozyme.

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Experimental Treatments

There are a number of experimental treatments for Pompe disease in various stages of development. These treatments are not yet approved, but they offer the potential for more options and better outcomes for Pompe patients in the future. These investigational treatments range from symptomatic therapies to those that may treat the underlying cause of Pompe.

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Non-drug Treatments

Most patients require supportive therapy to address the symptoms of Pompe disease, which include respiratory and cardiac problems, physical disability, and difficulty swallowing. Non-drug treatments such as physical therapy, occupational therapy, and speech therapy can help maintain patients’ quality of life, mobility, and communication skills for as long as possible.