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Metabolism, Inborn Errors

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Metabolism, Inborn Errors

Explore clinical trials studying Metabolism, Inborn Errors. Study availability and eligibility vary by location and protocol.

2,496 public trials 457 recruiting studies

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Metabolism, Inborn Errors trial results

Enrolling by Invitation

Theophylline for Treatment of Pseudohypoparathyroidism

NCT04240821

Conditions: Albright Hereditary Osteodystrophy, Bone Diseases, Bone Diseases, Metabolic, +10 more
Locations: Vanderbilt University Medical Center Nashville, Tennessee, United States

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Long-term Follow-up of Participants Dosed With an Investigational Gene Editing Therapy for Cardiovascular Disease

NCT06112327

Conditions: Arterial Occlusive Diseases, Arteriosclerosis, Atherosclerosis, +16 more
Locations: Clinical Study Center Auckland, New Zealand

Clinical Study Center Christchurch, New Zealand

Clinical Study Center London, United Kingdom

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Phase I/II Trial of Everolimus in Combination With Lonafarnib in Progeria

NCT02579044

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Laminopathies, +4 more
Locations: Boston Children's Hospital Boston, Massachusetts, United States

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Sirolimus for Leigh Syndrome

NCT06843811

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations: Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States

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Anderson-Fabry Disease Fitness Improvement Training: A-FAD-FIT

NCT07495410

Conditions: Behavior, Brain Diseases, Brain Diseases, Metabolic, +21 more
Locations: General University Hospital Prague, Czechia

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Improving Diagnosis and Clinical Management of Familial Hypercholesterolemia Through Integrated Machine Learning, Implementation Science, and Behavioral Economics

NCT05746247

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Familial Hypercholesterolemia, +9 more
Locations: University of Pennsylvania Health System Philadelphia, Pennsylvania, United States

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Digital Care for Holistic Health in Older Adults With Diabetes and Multimorbidity

NCT07310849

Conditions: Arthritis, Arthritis, Gouty, CKD, +23 more
Locations: Wugu District Public Health Center, New Taipei City, Wugu District, Taiwan

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Registry of Patients With Hypophosphatasia

NCT02306720

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Hypophosphatasia, +5 more
Locations: Research Site Centennial, Colorado, United States

Research Site Hartford, Connecticut, United States

Research Site Tampa, Florida, United States

Research Site Decatur, Georgia, United States

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ATTRACT-52: Primary Care Cardiac Amyloidosis Screening in Ordu, Turkey

NCT07238426

Conditions: Amyloid Neuropathies, Amyloid Neuropathies, Familial, Amyloidosis, +15 more
Locations: Ordu University Faculty of Medicine, Department of Cardiology Ordu, Turkey (Türkiye)

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Safety and Efficacy of AAV9. hMCOLN1co For Patients With Mucolipidosis Type IV

NCT07398872

Conditions: Bone Diseases, Bone Diseases, Metabolic, Brain Diseases, +15 more
Locations: Children's Hospital Zhejiang University School of Medicine Hangzhou, Zhejiang, China

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Metabolism, Inborn Errors

Metabolism, Inborn Errors trial results

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