Search clinical trials for: inborn errors of metabolism
Total 431502 results
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Inozyme PharmaNot yet recruitingGeneralized Arterial Calcification of Infancy | Autosomal Recessive Hypophosphatemic Rickets | Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency | ATP-Binding Cassette Subfamily C Member 6 Deficiency
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Eunice Kennedy Shriver National Institute of Child...RecruitingCHILD Syndrome | Smith Lemli Opitz Syndrome | Lathosterolosis | DesmosterolosisUnited States
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Tang-Du HospitalAir Force Military Medical University, ChinaNot yet recruiting
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Recordati Rare DiseasesTarget PharmaSolutions, Inc.RecruitingPropionic Acidemia | Methylmalonic Acidemia | HyperammonemiaUnited States
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First Affiliated Hospital Xi'an Jiaotong UniversitySimcere CompanyRecruitingCerebral Infarction | G6PD DeficiencyChina
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Taipei Veterans General Hospital, TaiwanNot yet recruitingGlycogen Storage Disease Type II
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National Human Genome Research Institute (NHGRI)RecruitingDiabetes | Undiagnosed Diseases | Inherited Metabolic Disorders | Batten's Disease | Tay SachsUnited States
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Massachusetts General HospitalEuropean Leukodystrophy AssociationRecruitingRestless Legs Syndrome | AdrenoleukodystrophyUnited States, Netherlands
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University of LausanneRecruiting
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Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyRecruitingPrimary Hyperoxaluria Type 3 | Primary Hyperoxaluria Type 2 | Primary Hyperoxaluria Type 1 | Primary HyperoxaluriaUnited States, Canada, Lebanon, Turkey, United Kingdom, Germany, Italy, Japan, Poland, Spain, United Arab Emirates
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Aspa TherapeuticsRecruitingCanavan DiseaseUnited States
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Hong Kong Children's HospitalRecruitingGlycogen Storage Disease Type IBHong Kong
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Alnylam PharmaceuticalsRecruitingPrimary Hyperoxaluria Type 1France, Spain, United States, Israel, Belgium, Switzerland, Germany, United Kingdom, Italy, Canada
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National Taiwan University HospitalRecruiting
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Duke UniversityRecruiting
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Stephen G. Kaler, MDRecruitingMenkes Disease | Occipital Horn SyndromeUnited States
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Viking Therapeutics, Inc.RecruitingAdrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)United States, France, Germany, Italy, United Kingdom
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National Institute of Diabetes and Digestive and...RecruitingPrimary Hyperparathyroidism | Pseudohypoparathyroidism | Parathyroid Cancer | Inheritable Bone Diseases | Multiple Endocrine Neoplasia, Type 1United States
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University Medical Centre LjubljanaNot yet recruitingPhenylketonurias | Nutritional and Metabolic Diseases
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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI); National Heart, Lung, and Blood Institute... and other collaboratorsRecruitingCongenital Amegakaryocytic Thrombocytopenia | Paroxysmal Nocturnal Hemoglobinuria | Shwachman-Diamond Syndrome | Bone Marrow Failure Syndrome | Congenital Pure Red Cell Aplasia | Hereditary Sideroblastic Anemia | Hematologic Neoplasm With Germline GATA2 Mutation | Hematologic Neoplasm With Germline... and other conditionsUnited States
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Second Affiliated Hospital, School of Medicine,...RecruitingClinical Features and OutcomeChina
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Second Affiliated Hospital, School of Medicine,...Recruiting
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Peking University Third HospitalNot yet recruitingFamilial Hypercholesterolemia
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Mackay Memorial HospitalSanofi Taiwan Co. LtdRecruiting
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Second Affiliated Hospital, School of Medicine,...RecruitingSingle Nucleotide Polymorphism | Hyperuricemia | Gout | Gut Microbiota | Uric Acid | Genetic Loci | FecesChina
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Eunice Kennedy Shriver National Institute of Child...RecruitingTurner Syndrome | Galactosemia | Ovarian Disfunction | Post-menarcheal Adolescents | Variations in Sex Characteristics | Differences in Sex DevelopmentUnited States
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Shenzhen People's HospitalRecruitingEvaluate the Efficacy and Safety of IBI306 in Subjects With Homozygous Familial HypercholesterolemiaLipid Metabolism Disorders | Familial Hypercholesterolemia - Homozygous | Proprotein Convertase Subtilisin/Kexin 9China
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Central Hospital, Nancy, FranceRecruitingPompe's Disease Juvenile OnsetFrance
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Vanderbilt University Medical CenterNational Heart, Lung, and Blood Institute (NHLBI)RecruitingFamilial HypercholesterolemiaUnited States
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Rigshospitalet, DenmarkDanish Research Center for Magnetic ResonanceRecruitingGlycogen Storage Disease | Pompe Disease (Late-onset) | McArdle DiseaseDenmark
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Children's Memorial Health Institute, PolandDepartment of Internal Medicine, Hypertension and Vascular Diseases, The...Not yet recruitingGlucose 6 Phosphatase DeficiencyPoland
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Assistance Publique - Hôpitaux de ParisRecruitingAdrenoleukodystrophy | Adrenomyeloneuropathy | Metachromatic Leukodystrophy | Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented GliaFrance
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Samsung Medical CenterNot yet recruitingSanfilippo Syndrome A
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SanofiRecruitingGlycogen Storage Disease Type IIBelgium, United States, China, Germany, Italy, Netherlands, Spain, Taiwan, United Kingdom
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Baylor College of MedicineChildren's National Research InstituteRecruitingOrnithine Transcarbamylase Deficiency | Urea Cycle Disorder | Carbamyl Phosphate Synthetase Deficiency | Argininosuccinic Aciduria | Hyperargininemia | Citrullinemia 1 | ARGI Deficiency | ASL Deficiency | ASS Deficiency | NAGS DeficiencyUnited States
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Massachusetts General HospitalAgios Pharmaceuticals, Inc.RecruitingAcute Myeloid Leukemia | Myelodysplastic Syndromes | Myeloproliferative Neoplasm | Myelodysplastic/Myeloproliferative Neoplasm | Clonal Cytopenia of Undetermined Significance | Pyruvate Kinase Deficiency | Pyruvate Kinase Deficiency Anemia | Hereditary Hemolytic Anemia | Clonal Myeloid Neoplasm | Other... and other conditionsUnited States
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Ain Shams UniversityRecruiting
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Children's Mercy Hospital Kansas CityRecruiting
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ModernaTX, Inc.RecruitingMethylmalonic AcidemiaUnited States, Spain, Canada, Netherlands, France, Australia, United Kingdom
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National Medical Research Center for Rehabilitation...Pirogov Russian National Research Medical University; DNKOM LLC; The Orenburg...RecruitingCardiovascular Diseases | Nervous System Diseases | Urologic Diseases | Diabetes | Neuromuscular Diseases | Hypercholesterolemia | Gout | Hyperbilirubinemia | Hyper IgE Syndrome | Hyper-LDL-cholesterolemia | Hyper Beta Lipoproteinemia | Hyper Eosinophilic SyndromeRussian Federation
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Chang Gung Memorial HospitalRecruitingNon-Phenylketonuric HyperphenylalaninemiaTaiwan
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University of Sao Paulo General HospitalPfizerRecruiting
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Shanghai Jiao Tong University School of MedicineRecruitingCongenital Adrenal Hyperplasia | Cushing Syndrome | Pheochromocytoma | Adrenal Incidentaloma | Adrenal Adenoma | Adrenal Carcinoma | Aldosteronism | HypoadrenalismChina
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University of California, San FranciscoRecruitingSphingolipidoses | Enzyme DeficiencyUnited States
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Massachusetts General HospitalNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)Recruiting
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Assistance Publique - Hôpitaux de ParisNot yet recruitingX-linked Hypophosphatemia (XLH)
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Cyclo Therapeutics, Inc.RecruitingNiemann-Pick Disease, Type C1United States, Spain, Israel, Taiwan, Brazil, Germany, Australia, Poland, Turkey, Italy, United Kingdom
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University Health Network, TorontoUniversity of Colorado, Denver; Mayo Clinic; Icahn School of Medicine at Mount... and other collaboratorsRecruiting
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Akcea TherapeuticsUnited BioSource, LLCRecruitingHereditary Transthyretin Amyloidosis With PolyneuropthyUnited States, France, Greece, Italy, Spain, Canada, Germany, Portugal
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Ionis Pharmaceuticals, Inc.RecruitingAlexander DiseaseUnited States, United Kingdom, Australia, Canada, Israel, Italy, Japan, Netherlands