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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

A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04

NCT05146830

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystinosis, Genetic Diseases, Inborn, +4 more
Locations: University of California San Diego La Jolla, California, United States

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Enrolling by Invitation

Cardioprotective and Mental Health Benefits of the MIND Diet Combined With Forest Bathing

NCT06222632

Conditions: Anxiety Disorders, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, +11 more
Locations: 16/F, Ma Kam Chan Memorial Building, 31 Wylie Road, Homantin, Hong Kong

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Enrolling by Invitation

Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE

NCT05492799

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations: UCSF Benioff Children's Hospital Oakland Oakland, California, United States

UPMC Children's Hospital Pittburgh Pittsburgh, Pennsylvania, United States

Fundación Cardio Infantil - Instituto de Cardiología Bogotá, Colombia

University Medical Center Hamburg-Eppendorf Hamburg, Germany

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Enrolling by Invitation

Evaluation of a Digiphysical Screening Method to Identify and Diagnose Familial Hypercholesterolemia

NCT04929457

Conditions: Arterial Occlusive Diseases, Arteriosclerosis, Atherosclerosis, +27 more
Locations: Karolinska university hospital Stockholm, Sweden

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Enrolling by Invitation

Long-Term Follow Up Study of Subjects Previously Administered HMI 102

NCT04348708

Conditions: Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +11 more
Locations: Boston Children's Hospital Boston, Massachusetts, United States

Icahn School of Medicine at Mount Sinai New York, United States

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Not Yet Recruiting

Study of LNP.UCD.ABE in Patients With Urea Cycle Disorders

NCT07667387

Conditions: Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +11 more
Locations: Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States

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Not Yet Recruiting

Diazoxide in the Treatment of Type 1 Glycogenosis

NCT07739394

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +5 more
Locations: Not specified

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Not Yet Recruiting

ATR 1072 in Participants With PRKAG2 Syndrome

NCT07738107

Conditions: Arrhythmias, Cardiac, Carbohydrate Metabolism, Inborn Errors, Cardiac Conduction System Disease, +20 more
Locations: Not specified

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Not Yet Recruiting

Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)

NCT07740512

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +24 more
Locations: Not specified

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Not Yet Recruiting

Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B)

NCT07579910

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations: Not specified

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Metabolism, Inborn Errors

Metabolism, Inborn Errors trial results

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