Search clinical trials for: inborn errors
Total 15908 results
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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
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Jiangsu HengRui Medicine Co., Ltd.Not yet recruitingPrimary Gout With Hyperuricemia in Adults
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Ultragenyx Pharmaceutical IncRecruitingOTC DeficiencyUnited States, Portugal, Italy, Spain, United Kingdom, Japan, Canada, Germany, France, Argentina, Netherlands, Brazil
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Freeline TherapeuticsRecruitingGaucher Disease, Type 1Spain, Israel, United States, United Kingdom, Brazil, Germany, Paraguay
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The Affiliated Hospital of Qingdao UniversityRecruiting
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Sun Yat-Sen Memorial Hospital of Sun Yat-Sen UniversityRecruiting
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ModernaTX, Inc.RecruitingMethylmalonic AcidemiaUnited States, Canada, Netherlands, United Kingdom
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Nemours Children's ClinicEunice Kennedy Shriver National Institute of Child Health and Human Development...RecruitingMucopolysaccharidosis IV Type A | Morquio A Syndrome | MPS IVAUnited States
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SanofiRecruitingFabry DiseaseItaly, United States, Austria, China, Czechia, Denmark, France, Germany, Greece, Japan, Korea, Republic of, Netherlands, Norway, Spain, Taiwan, Turkey, United Kingdom, Canada, Poland
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LMU KlinikumRecruitingPompe Disease (Late-onset) | FSHD | Spinal Muscular Atrophy Type 3 | Inclusion Body MyositisGermany
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Great Ormond Street Hospital for Children NHS Foundation...National Institute for Health Research, United Kingdom; The Freya FoundationRecruitingPyruvate Dehydrogenase Complex Deficiency | Pyruvate Dehydrogenase E1 Alpha Deficiency | Pyruvate Dehydrogenase E1-Beta Deficiency | Pyruvate Dehydrogenase E2 Deficiency | Pyruvate Dehydrogenase Phosphatase DeficiencyUnited Kingdom
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Shaare Zedek Medical CenterRecruiting
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OrphalanErgomedRecruitingWilson's DiseaseSpain, United Kingdom, Belgium, Germany, Poland, France, Saudi Arabia
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Essentia HealthSt. Luke's Hospital of Duluth; WHITESIDE Institute for Clinical ResearchRecruitingFamilial Hypercholesterolemia | Genetic Disease | Cholesterol, ElevatedUnited States
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AlexionRecruiting
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Fondation Ophtalmologique Adolphe de RothschildRecruiting
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Neurogene Inc.RecruitingNeuronal Ceroid Lipofuscinosis CLN5United States, United Kingdom
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Manchester University NHS Foundation TrustRecruitingAdrenal Hyperplasia, CongenitalUnited Kingdom
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SanofiRecruitingGaucher's Disease Type IIIUnited States, Germany, Hungary, France, China, Japan, Canada, Argentina, Italy, Turkey
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University of British ColumbiaVancouver Coastal Health Research Institute; Genome British ColumbiaRecruitingAcute Coronary Syndrome | Familial Hypercholesterolemia | STEMI | NSTEMI - Non-ST Segment Elevation MI | Familial Hypercholesterolemia - Heterozygous | Familial Hypercholesterolemia Due to Genetic Defect of Apolipoprotein B | Familial Hypercholesterolemia Due to Heterozygous LDL Receptor Mutation and other conditionsCanada
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Capital Medical UniversityRecruitingCerebral Amyloid AngiopathyChina
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Hospices Civils de LyonRecruitingPrimary Intestinal Hypocholesterolemia | Abetalipoproteinemia | Chylomicron Retention DiseaseFrance
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AO GENERIUMRecruitingMetabolic Diseases | Mucopolysaccharidosis Type IIRussian Federation
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SanofiRecruitingFabry DiseaseChina, Finland, Germany, Norway, United Kingdom, United States, Denmark, Austria, Greece, Romania, Argentina, Brazil, Canada, Japan, Mexico, Poland, Italy, France, Turkey, Australia, Switzerland
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Institut de Myologie, FranceRecruitingMuscular Dystrophies | Mitochondrial Myopathies | Glycogen Storage Disease | Idiopathic Inflammatory Myopathies | Congenital MyopathyFrance
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Institut de Myologie, FranceRecruitingMuscular Dystrophies | Mitochondrial Myopathies | Glycogen Storage Disease | Idiopathic Inflammatory Myopathies | Congenital MyopathyFrance
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Poxel SANot yet recruiting
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University of Sao Paulo General HospitalRecruitingMicrobial Colonization | Amyloidosis, Hereditary, Transthyretin-RelatedBrazil
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Fondation Ophtalmologique Adolphe de RothschildRecruiting
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Laval UniversityUniversité de Montréal; Centre de Recheche du Centre Hospitalier Université...RecruitingCardiovascular Diseases | Familial Hypercholesterolemia | Cholesterol, Elevated | Nutrition, Healthy | Cholesterol; Metabolic DisorderCanada
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Kyowa Kirin Pharmaceutical Development LtdRecruitingX-Linked HypophosphatemiaFrance, Netherlands, United Kingdom
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Agios Pharmaceuticals, Inc.RecruitingPediatric Pyruvate Kinase Deficiency | Pediatric Hemolytic AnemiaUnited States, Spain, Germany, Netherlands, Canada, France, Israel, Switzerland, Italy
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PTC TherapeuticsRecruitingPhenylketonuriaUnited States, Germany, Spain, Australia, Brazil, United Kingdom, Denmark, Mexico, Portugal, Turkey, Canada, Georgia, Italy, Netherlands, Japan
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Lovisenberg Diakonale HospitalUniversity of BergenNot yet recruitingIrritable Bowel Syndrome | Functional Gastrointestinal Disorders | Sucrase Isomaltase Deficiency | Sucrose Intolerance Due to Sucrase-Isomaltase Deficiency | Carbohydrate; Malabsorption
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Poxel SANot yet recruiting
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ModernaTX, Inc.RecruitingPropionic AcidemiaUnited States, Canada, United Kingdom
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Spruce BiosciencesRecruitingCongenital Adrenal Hyperplasia | 21-OHDUnited States