Study could offer hope to Pompe disease patients

Pompe disease is a rare genetic disorder that disables heart and skeletal muscles and can lead to early death if untreated. The only available treatment for the disease is enzyme replacement therapy that must be injected regularly, sometimes every few days, for life. The treatment can cost hundreds of thousands of dollars a year. Researchers have now developed a method that could make enzyme replacement therapy more efficient, less expensive and less frequent.

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