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Recruiting

Danon Disease Natural History Study

NCT06214507

Conditions
Carbohydrate Metabolism, Inborn Errors, Cardiomyopathies, Cardiovascular Diseases, +15 more
Locations
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • University of California, San Diego La Jolla, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • University of Florida College of Medicine Gainesville, Florida, United States
View all 15 locations
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Recruiting

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

NCT07282847

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations
  • Barrow Neurological Institute Phoenix, Arizona, United States
  • University of California, Irvine (UCI) Irvine, California, United States
  • Stanford Neuroscience Health Center Palo Alto, California, United States
  • NYU Langone New York, United States
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Recruiting

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

NCT06636383

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +8 more
Locations
  • Children's Hospital of Orange County Orange, California, United States
  • Children's Hospital Colorado Denver, Colorado, United States
  • University of Connecticut Health Center Hartford, Connecticut, United States
  • University of Michigan Ann Arbor, Michigan, United States
View all 19 locations
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Recruiting

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

NCT07354724

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • University of California-Irvine Irvine, California, United States
  • Duke University School of Medicine - Early Phase Research Unit Durham, North Carolina, United States
  • The Lysosomal & Rare Disorders Research & Treatment Center Fairfax, Virginia, United States
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Recruiting

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

NCT07123155

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
Locations
  • University of California - Irvine Medical Center Irvine, California, United States
  • University of Florida (UF) - Gainesville Gainesville, Florida, United States
  • Emory University Hospital Atlanta, Georgia, United States
  • Washington University in St. Louis St Louis, Missouri, United States
View all 28 locations
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Recruiting

Pompe Disease Registry Protocol

NCT00231400

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • University of Alabama at Birmingham- Site Number : 840106 Birmingham, Alabama, United States
  • Barrow Neurological Institute- Site Number : 840087 Phoenix, Arizona, United States
  • Phoenix Children's Hospital- Site Number : 840003 Phoenix, Arizona, United States
  • University of Arizona- Site Number : 840015 Tucson, Arizona, United States
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Recruiting

Dietary Treatment Strategies and Metabolic Control in Glycogen Storage Disease Type I

NCT06852612

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +5 more
Locations
  • Department of Diabetes, Endocrinology, Nutritional Medicine and Metabolism, Inselspital, Bern University Hospital Bern, Switzerland
  • Department of Endocrinology, Diabetology and Clinical Nutrition, University Hospital Zurich Zurich, Switzerland
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Recruiting

GSD VI and GSD IX Natural History

NCT04454216

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GLYCOGEN STORAGE DISEASE IXa1, +13 more
Locations
  • Duke University Durham, North Carolina, United States
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Recruiting

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

NCT06092034

Conditions
Aortic Stenosis, Subvalvular, Aortic Valve Disease, Aortic Valve Stenosis, +26 more
Locations
  • University of California, San Diego La Jolla, California, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
  • Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
  • German Heart Center Munich Munich, Germany
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Recruiting

Pompe Pregnancy Sub-Registry

NCT00567073

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Barrow Neurol Group- Site Number : 840087 Phoenix, Arizona, United States
  • University of Florida Pediatrics Genetics- Site Number : 157138 Jacksonville, Florida, United States
  • Emory University School Of Medicine- Site Number : 840060 Atlanta, Georgia, United States
  • Indianapolis University School of Medicine- Site Number : 840027 Indianapolis, Indiana, United States
View all 35 locations
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Recruiting

A Global Prospective Observational Registry of Patients With Pompe Disease

NCT06121011

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • University of Arkansas Medical Science Little Rock, Arkansas, United States
  • University of California Irvine Irvine, California, United States
  • Wolfson Children's Hospital Jacksonville, Florida, United States
  • Emory University Atlanta, Georgia, United States
View all 41 locations
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