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Pompe Disease

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Pompe Disease

Explore clinical trials studying Pompe Disease. Study availability and eligibility vary by location and protocol.

54 public trials 9 recruiting studies

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Pompe Disease trial results

Active, Not Recruiting

Safety and Efficacy of Avalglucosidase Alfa in Patients With Non-classic Pompe Disease Aged ≥ 5 Years

NCT07652814

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
Locations: Erasmus MC Rotterdam, Netherlands

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Active, Not Recruiting

Developing a Management Approach for Patients With "Late-Onset" Pompe Disease

NCT03694561

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations: Duke University Durham, North Carolina, United States

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Active, Not Recruiting

Novel Metabolic Muscular Biomarkers in Pompe Disease - a Non-invasive Magnetic Resonance Exploratory Pilot Study.

NCT05943678

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations: Medical University of Vienna Vienna, State of Vienna, Austria

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Active, Not Recruiting

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

NCT04093349

Conditions: Acid Maltase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +17 more
Locations: Barrow Neurological Institute Phoenix, Arizona, United States

University of California Irvine Health Orange, California, United States

Emory University School of Medicine Atlanta, Georgia, United States

University of Kansas Medical Center Research Institute Kansas City, Kansas, United States

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