This Drug Could be a New Treatment for Pompe Disease

According to a story from Science Trends, a recent trial of an experimental treatment demonstrates that it could be a potential treatment for Pompe disease. There are very limited treatment options for Pompe disease, and there is a dire need for a greater variety of treatment options as well as options that are more effective.

About Pompe Disease

Pompe disease, which is also known as glycogen storage disease type II, is a genetic, metabolic disorder. This disease can cause damage to the nerves and muscles throughout the body, and is the result of the excessive buildup of glycogen in the cellular lysosome. This occurs because of the deficiency of the certain enzyme. The disease is the result of a genetic mutation that appears on chromosome 17. Symptoms of Pompe disease vary depending on when it appears. They can include poor growth, trouble feeding, enlarged heart, poor muscle tone, muscle weakness, and breathing problems. There is also a late onset form that mostly differs by the absence of heart abnormalities. The primary treatment for Pompe disease is enzyme replacement. While this treatment can improve symptoms and survival, a high dosage is necessary and it primarily only halts disease progression. To learn more about Pompe disease, click here.

About The Study

A recent clinical trial of the experimental therapy clenbuterol displayed the potential benefits of this drug. The medication was able to reduce the concentration of glycogen stored in the body and caused improvements in the six minuted walk test, which is a common measurement tool for Pompe disease treatment. The research showed that the use of clenbuterol in combination with enzyme replacement was able to substantially improve muscle strength in comparison to enzyme replacement alone.

An Advancement in Treatment?

Improvements in muscle strength also included the breathing muscles, a significant benefit, and trials have shown clenbuterol to greatly enhance the effectiveness of enzyme replacement therapy in both the infantile and late onset form of the disease. Overall, the results for clenbuterol are looking quite promising so far, although more clinical trials will be necessary.

The original research study can be found here and was originally published in the journal Molecular Therapy.


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