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.

Show More Content Like This

More Clinical Research/Studies Content

How do I give money to help research in Pompe disease?

How do I give money to help research in Pompe disease?

There are several support groups that support individuals with Pompe disease and raise money for research into better treatments. One organization is called the Acid Maltase Deficiency Association (AMDA). Another is the Muscular Dystrophy Association (MDA) which support people with many different muscle diseases, including Pompe disease. The Genetic Alliance is an umbrella organization which has information about support groups for many different organizations, including Pompe Disease. You can look up information about other groups at the Genetic Alliance website.

We use cookies to ensure that we give you the best experience on our website. By continuing to browse this site, you are agreeing to our use of cookies.

Continue Find out more about our use of cookies and similar technology

This content comes from a hidden element on this page.

The inline option preserves bound JavaScript events and changes, and it puts the content back where it came from when it is closed.

Remember Me