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Metabolism, Inborn Errors

Clinical trial condition

Metabolism, Inborn Errors

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

2,493 public trials 456 recruiting studies

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Metabolism, Inborn Errors trial results

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

Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA

NCT06095388

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations: Universitätsklinikum Hamburg-Eppendorf Hamburg, Germany

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Active, Not Recruiting

Prospective Evaluation of NfL as a Biomarker in ATTRv

NCT05879874

Conditions: Amyloidosis, Amyloidosis, Familial, Amyloidosis, Hereditary, +10 more
Locations: Fondazione Policlinico Gemelli IRCCS Rome, Italy

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Carbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage Diseases

NCT04929002

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations: Neuromuscular Research Unit, 8077 Copenhagen, Copenhange, Denmark

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A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

NCT04251026

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +22 more
Locations: UCSF Benioff Children's Hospital Oakland, California, United States

Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States

UNC Children's Research Institute Chapel Hill, North Carolina, United States

UPMC | Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States

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Active, Not Recruiting

Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae

NCT03285425

Conditions: Batten Disease, CLN6, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +11 more
Locations: Nationwide Children's Hospital Columbus, Ohio, United States

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An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II

NCT03708965

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
Locations: Grupo de Pesquisa Clínica em Genética Médica - HCPA Porto Alegre, Brazil

Igeim - Unifesp São Paulo, Brazil

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Active, Not Recruiting

An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II

NCT04348136

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
Locations: Fukui Clinical site Fukui, Japan

Fukuoka Clinical site 2 Fukuoka, Japan

Fukuoka Clinical site Fukuoka, Japan

Gifu Clinical site Gifu, Japan

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Long Term Follow-Up for Safety of AVR-RD-02

NCT06488261

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations: University of Iowa Hospitals and Clinics Iowa City, Iowa, United States

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Burden of Disease and Functional Impairment in XLH

NCT04946409

Conditions: Avitaminosis, Bone Diseases, Bone Diseases, Metabolic, +26 more
Locations: Orthopedic Center for Musculoskeletal Research, Orthopedic Department, University of Wuerzburg Würzburg, Germany

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Metabolism, Inborn Errors

Metabolism, Inborn Errors trial results

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