Pompe Disease Clinical Trial
Pompe Disease is a genetic disorder that causes glycogen to build up in your body. As the enzyme builds up on your muscles and organs, it causes them to deteriorate. It often results in weak muscles, an enlarged heart and liver, and breathing difficulties, making it a dangerous condition.
In this trial we will study a new combination treatment (an investigational enzyme replacement therapy and oral drug ) for Pompe Disease in adolescents.
Who can take part in the study?
We are conducting a new combination treatment for adolescents diagnosed with Pompe Disease.
Age
12-18 years old
Key Inclusion and Exclusion Criteria:
- Diagnosis of Late Onset Pompe Disease
- May be ERT naïve or experienced
- You are able to walk at least 75 meters (82 yards)
Patient Commitment Duration:
About 60 weeks with the option to enroll in an open-label extension study
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