Pompe disease

Clinical Research/Studies

Is there clinical research going on for Pompe disease?

There are several clinical trials ongoing for Pompe disease. Alglucosidase alfa (Lumizyme®), an enzyme replacement therapy, is currently the only clinically available treatment for Pompe disease (PD). There are multiple ongoing clinical research projects to better understand Pompe disease and other medications. There are clinical trials on the horizon for other possible treatments for Pompe disease such as combination therapy and gene therapy. All clinical trials must be registered on clinicaltrials.gov. If you are interested in enrolling in research, visit the site regularly as studies change enrollment status often.

Antidote also offers a handy tool to find clinical trials.

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