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Brain Diseases, Metabolic, Inborn

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Brain Diseases, Metabolic, Inborn

Explore clinical trials studying Brain Diseases, Metabolic, Inborn. Study availability and eligibility vary by location and protocol.

949 public trials 197 recruiting studies

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Brain Diseases, Metabolic, Inborn trial results

Not Yet Recruiting

Phase I/II Clinical Study to Evaluate the Safety, Tolerability and Efficacy of LY-M003 Injection in Adult Patients With Wilson's Disease

NCT07641140

Conditions: Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations: The First Affiliated Hospital, Zhejiang University School of Medicine Hangzhou, Zhejiang, China

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Not Yet Recruiting

PKU Microtablets Case Studies

NCT07671859

Conditions: Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations: University Hospitals Bristol and Weston NHS Foundation Trust Bristol, United Kingdom

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A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)

NCT06792500

Conditions: Acid-Base Imbalance, Acidosis, Acidosis, Lactic, +43 more
Locations: Children's National Hospital Washington D.C., District of Columbia, United States

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Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.

NCT07465718

Conditions: Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations: University of Colorado Anschutz School of Medicine Denver, Colorado, United States

Yale University School of Medicine New Haven, Connecticut, United States

University of Michigan Medical Centre Ann Arbor, Michigan, United States

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A Phase I/II Clinical Study to Evaluate the Safety and Efficacy of VGN-R08b in Patients With Type III Gaucher's Disease

NCT07603050

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations: Not specified

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Quality of Life in Adults With Untreated Fabry Disease in Sweden (QoLUF)

NCT07560956

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +23 more
Locations: Sahlgrenska University Hospital Gothenburg, Sweden

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PheCheck™ Validation Study

NCT07551921

Conditions: Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations: Not specified

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Not Yet Recruiting

Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial

NCT07479953

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
Locations: Not specified

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Not Yet Recruiting

A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion

NCT05669729

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations: Not specified

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Not Yet Recruiting

Care Pathway for Patients With Fabry's Disease (Fabry-PATH)

NCT07485660

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Service de Cardiologie - 1 Avenue du Professeur Jean Poulhès Toulouse, Occitanie, France

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Brain Diseases, Metabolic, Inborn

Brain Diseases, Metabolic, Inborn trial results

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