ClinicaliQ Trial Snapshot
- A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease — Active Not Recruiting • Non-phase study • Gastroenterology • NCT06150820.
- Study measuring antibody and biomarker levels in blood of adults with late-onset Pompe disease to assess gene therapy readiness.
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What This Trial Is Studying
Pompe disease is a genetic condition which causes muscle weakness over time. People with Pompe disease have a faulty gene that makes an enzyme called acid alpha-glucosidase (or GAA). This enzyme breaks down a type of sugar called glycogen. Without this enzyme, there is a build-up of glycogen in the cells of the body. This causes muscle weakness and other symptoms. Pompe disease can happen at any age, but in late-onset Pompe disease, symptoms generally start from 12 months old onwards. The standard treatment for people with Pompe disease is…