Search clinical trials for: Inborn errors of metabolism
Total 433814 results
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University of MinnesotaRecruitingGaucher Disease, Type 3United States
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LG ChemRecruitingHyperuricemia | Gout | Gout Flare | TophiUnited States
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BioMarin PharmaceuticalSyneos HealthRecruitingPhenylketonuria, MaternalGermany, United States, Italy
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Massachusetts General HospitalPfizer; Akcea Therapeutics; Alnylam Pharmaceuticals; Eidos Therapeutics, a BridgeBio...RecruitingHeart Failure | Amyloidosis | Heart Failure, Diastolic | Transthyretin AmyloidosisUnited States
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University of ThessalyNot yet recruitingOxidative Stress | Carbohydrate Metabolism Disorder | G6PD Deficiency
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Marcello ArcaPfizer; Göteborg University; University of HelsinkiNot yet recruitingFamilial Hypobetalipoproteinemia | Familial Hypobetalipoproteinemia - Heterozygous Form
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Seventh Medical Center of PLA General HospitalGeneCradle Therapeutics, IncRecruiting
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LMU KlinikumUniversity of Pisa; German Federal Ministry of Education and Research; European...RecruitingMitochondrial Diseases | MDS | Mitochondrial Myopathies | MELAS Syndrome | MIDD | Kearns-Sayre Syndrome | MERRF Syndrome | Barth Syndrome | Leigh Syndrome | MNGIE | LHON | Pearson Syndrome | NARP Syndrome | Coenzyme Q10 Deficiency | SANDO | SCAE | MIRAS | CPEOAustria, Germany, Italy
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Glycomine, Inc.Recruiting24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Participants With PMM2-CDGPmm2-CDGSpain, Australia, United States
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Assistance Publique - Hôpitaux de ParisRecruiting
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Uskudar UniversityRecruitingLesch-Nyhan SyndromeTurkey
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Assistance Publique - Hôpitaux de ParisCystinosis Research FoundationRecruiting
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New York Stem Cell Foundation Research InstituteSilverstein FoundationRecruitingHealthy | Parkinson Disease | Gaucher Disease | GBA Gene MutationUnited States
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Arcturus Therapeutics, Inc.RecruitingOTC Deficiency | Ornithine Transcarbamylase Deficiency | OTCDSpain, Belgium, United Kingdom, Sweden
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Sanguine BiosciencesDenali Therapeutics Inc.RecruitingMucopolysaccharidosis III-AUnited States
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Jiangsu HengRui Medicine Co., Ltd.Not yet recruitingPrimary Gout and Hyperuricemia
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Alice BertainaCalifornia Institute for Regenerative Medicine (CIRM)RecruitingCKD Stage 4 | Cystinosis | FSGS | SLE Nephritis | SIODUnited States
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University of EdinburghNot yet recruiting
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Chinese University of Hong KongRecruiting
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Leiden University Medical CenterRecruitingCognitive Decline | Cerebral Amyloid Angiopathy | Intracerebral Hemorrhage | Hypertensive Hemorrhage, IntracranialNetherlands
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TakedaTakeda Development Center Americas, Inc.RecruitingHunter Syndrome | Mucopolysaccharidosis (MPS)United States
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Fondation Ophtalmologique Adolphe de RothschildRecruiting
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University of Texas Southwestern Medical CenterNational Heart, Lung, and Blood Institute (NHLBI); Columbia University; The Cleveland... and other collaboratorsRecruitingAmyloidosis, Familial | Amyloidosis, Hereditary | Amyloidosis Cardiac | Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin Gene MutationUnited States
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APR Applied Pharma Research s.a.RecruitingPhenylketonuriasUnited Kingdom
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Prevail TherapeuticsEli Lilly and CompanyRecruitingGaucher Disease | Gaucher Disease, Type 1United States, Spain, Germany
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QOL Medical, LLCRecruitingCongenital Sucrase-Isomaltase Deficiency | CSID | Sucrase Isomaltase DeficiencyUnited States
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National Taiwan University HospitalRecruitingStroke | Magnetic Resonance Imaging | Fabry Disease | Cerebral Small Vessel Diseases | Cadasil | HTRA1-Related Autosomal Dominant Cerebral Angiopathy | COL4A1-Related Brain Small Vessel Disease With Haemorrhage | Next-generation SequencingTaiwan
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Merit UniversityNot yet recruiting
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CANbridge (Suzhou) Bio-pharma Co., Ltd.RecruitingGaucher Disease, Type 1 | Gaucher Disease, Type 3China
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Centre for Analytical Biochemistry and Biomedical...University of GiessenRecruiting
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Hugo W. Moser Research Institute at Kennedy Krieger...RecruitingNeurodegenerative Diseases | Ataxia | Cerebellar Ataxia | Leukodystrophy | Adrenomyeloneuropathy | LBSL | Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation | Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)United States
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University of NebraskaRecruiting
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Fondation Ophtalmologique Adolphe de RothschildRecruiting
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Duke UniversityGenzyme, a Sanofi CompanyRecruitingLate-Onset Pompe Disease | Lysosomal DiseaseUnited States
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GC Biopharma CorpRecruitingMucopolysaccharidosis II | Hunter SyndromeKorea, Republic of
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University of Roma La SapienzaAlexion PharmaceuticalsRecruitingNon-Alcoholic Fatty Liver DiseaseItaly
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Academisch Medisch Centrum - Universiteit van Amsterdam...RecruitingFabry Disease | Fabry Disease, Cardiac VariantNetherlands
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University Children's Hospital, ZurichRecruitingInborn Errors of Metabolism | TransitionSwitzerland, Austria
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University of MilanRecruitingCOPD | Dyspnea | ArgininemiaItaly
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Universidade do PortoCentro Hospitalar Universitário São João, E.P.E. (CHUSJ); Centro de Investigação... and other collaboratorsRecruitingCarpal Tunnel Syndrome | Transthyretin AmyloidosisPortugal
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Universidade do PortoHospital da Luz, Lisboa; Hospital Pedro Hispano; Centro Hospitalar de Leiria; Centro... and other collaboratorsRecruitingFabry DiseasePortugal
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Eva Morava-KoziczNot yet recruitingSLC35A2-CDG - Solute Carrier Family 35 Member A2 Congenital Disorder of GlycosylationUnited States
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Eva Morava-KoziczChildren's Hospital of Philadelphia; Seattle Children's HospitalNot yet recruiting
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Eva Morava-KoziczNot yet recruitingPGM1-CDG - Phosphoglucomutase 1-Related Congenital Disorder of GlycosylationUnited States
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SwanBio Therapeutics, Inc.RecruitingAMN | AMN Gene Mutation | X-ALDNetherlands, United States
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Denali Therapeutics Inc.RecruitingMucopolysaccharidosis IIUnited States, Spain, United Kingdom, Czechia, France, Canada, Italy, Belgium, Netherlands, Germany, Argentina, Sweden, Turkey, Brazil, Colombia
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Oslo University HospitalUniversity of OsloRecruitingFamilial HypercholesterolemiaNorway
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SanofiRecruitingAcid Sphingomyelinase Deficiency (ASMD)France
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Manchester University NHS Foundation TrustOrchard TherapeuticsRecruitingMucopolysaccharidosesUnited Kingdom
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University of Maryland, BaltimoreRecruitingFamilial Hypercholesterolemia | Long QT Syndrome | Genetic Testing | EthicsUnited States