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Search clinical trials for: Methylmalonic Aciduria
Total 49 results
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West Kazakhstan Medical UniversityRecruitingOrnithine Transcarbamylase Deficiency | Biotinidase Deficiency | Citrullinemia | Glutaric Acidemia Type II | Argininosuccinic Aciduria | Maple Syrup Urine Disease | Primary Carnitine Deficiency | Homocystinuria | Carnitine Palmitoyltransferase II Deficiency | Arginase Deficiency | Very Long-chain Acyl-CoA... and other conditionsKazakhstan
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Centre Hospitalier Universitaire de LiegeSanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaboratorsRecruitingCongenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditionsBelgium
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ModernaTX, Inc.RecruitingMethylmalonic AcidemiaUnited States, Canada, Netherlands, United Kingdom
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ModernaTX, Inc.RecruitingMethylmalonic AcidemiaUnited States, Spain, Canada, Netherlands, France, Australia, United Kingdom
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Fondazione I.R.C.C.S. Istituto Neurologico Carlo...RecruitingNeurologic Disorder | Rare Diseases | Metabolic Disease | Inherited Disease | Undiagnosed Disease | Neuro-Degenerative DiseaseItaly
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RTI InternationalEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationPrimary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditionsUnited States
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Emma Marie Caroline SlackUniversity Children's HospitalCompletedInborn Errors of Metabolism | Propionic Aciduria | Methylmalonic Aciduria | Urea Cycle DisorderSwitzerland
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Bambino Gesù Hospital and Research InstituteCatholic University of the Sacred HeartCompletedGenetic Disease | Retinopathy | Methylmalonic Aciduria and Homocystinuria,Cblc TypeItaly
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Uta Lichter-KoneckiMedical College of Wisconsin; Children's National Research InstituteCompletedUrea Cycle Disorders | Organic AcidemiasUnited States
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McGill University Health Centre/Research Institute...UnknownMethylmalonic Acidemia | Malonic AciduriaCanada
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Shanghai Vitalgen BioPharma Co., Ltd.RecruitingGlutaric Acidemia Type I | Glutaric Aciduria Type IChina
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National Human Genome Research Institute (NHGRI)RecruitingMetabolic Disease | Purine-Pyrimidine Metabolism | AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2 | UNG, OMIM *191525, Hyper-IgM Syndrome 5 | NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency | UMPS, OMIM *613891, Orotic Aciduria | DHODH, OMIM *126064,... and other conditionsUnited States
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Fundació EurecatLaboratorio Echevarne; Health Tech Bio Actives, S.L.U.RecruitingEffect of Methylcobalamin and Cyanocobalamin Consumption on Vitamin B12 Nutritional Status (NORMB12)Vitamin B12 Nutritional DeficiencySpain
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ModernaTX, Inc.RecruitingPropionic AcidemiaUnited States, Canada, United Kingdom
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Recordati Rare DiseasesTarget PharmaSolutions, Inc.RecruitingPropionic Acidemia | Methylmalonic Acidemia | HyperammonemiaUnited States
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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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Baylor College of MedicineChildren's Hospital of Philadelphia; Children's Hospital Colorado; Seattle Children... and other collaboratorsRecruitingOrnithine Transcarbamylase Deficiency | Urea Cycle Disorder | Argininosuccinic Aciduria | Hyperargininemia | Citrullinemia 1 | ARGI Deficiency | ASL Deficiency | ASS DeficiencyUnited States
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Recordati Rare DiseasesRecruitingPropionic Acidemia | Methylmalonic AcidemiaFrance, Spain, Italy, United Kingdom, Norway, Germany, Sweden
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ModernaTX, Inc.RecruitingPropionic AcidemiaUnited States, Canada, United Kingdom
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National Human Genome Research Institute (NHGRI)RecruitingInborn Errors of Metabolism | Methylmalonic Acidemia | Organic AcidemiaUnited States
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LogicBio Therapeutics, IncAlexion Pharmaceuticals, Inc.TerminatedMethylmalonic AcidemiaUnited States
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HemoShear TherapeuticsTerminatedPropionic Acidemia | Methylmalonic AcidemiaUnited States, Saudi Arabia, Australia
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HemoShear TherapeuticsPrometrika, LLC; AllStripes Research Inc.; Genome MedicalTerminatedPropionic Acidemia | Methylmalonic AcidemiaUnited States, Canada, United Kingdom
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CoA Therapeutics, Inc., a BridgeBio companyTerminatedHealthy Volunteers | Propionic Acidemia | Methylmalonic Acidemia | Organic AcidemiaUnited States
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LogicBio Therapeutics, IncAlexion Pharmaceuticals, Inc.Active, not recruitingMethylmalonic AcidemiaUnited States
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Selecta Biosciences, Inc.National Human Genome Research Institute (NHGRI)SuspendedMethylmalonic Acidemia (MMA)United States
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Target PharmaSolutions, Inc.Recordati Rare DiseasesEnrolling by invitationPropionic Acidemia | Methylmalonic Acidemia | HyperammonemiaUnited States
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Baylor College of MedicineRare Diseases Clinical Research Network; Neogenis LaboratoriesCompletedUrea Cycle Disorders, Inborn | Urea Cycle Disorder | Argininosuccinic Aciduria | Argininosuccinate Lyase DeficiencyUnited States
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KU LeuvenCompleted
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ModernaTX, Inc.CompletedPropionic Acidemia | Methylmalonic AcidemiaUnited States, United Kingdom, Spain, France
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Mendel TuchmanChildren's Hospital of Philadelphia; University of California, Los Angeles; Icahn... and other collaboratorsCompletedMethylmalonic Acidemia | Carbamoyl-Phosphate Synthase I Deficiency Disease | Propionic Acidemia, Type I and/or Type II | Ornithine Carbamoyltransferase DeficiencyUnited States
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University of UlsterUniversity of Bergen; University of Dublin, Trinity CollegeCompleted
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Indiana UniversityCompletedKidney CalculiUnited States
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ModernaTX, Inc.WithdrawnMetabolism, Inborn Errors | Methylmalonic Acidemia (MMA)
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University Children's Hospital, ZurichCompletedPhenylketonurias | OTC Deficiency | Inborn Errors of Metabolism | Urea Cycle Disorder | Maple Syrup Urine Disease | Methylmalonicacidemia | Aminoacidopathy | Patient Reported Outcome MeasurementsSwitzerland
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PTC TherapeuticsGenzyme, a Sanofi CompanyTerminatedAmino Acid Metabolism, Inborn ErrorsFrance, United Kingdom, Italy, Belgium, Germany, Switzerland
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National Taiwan University HospitalUnknownPropionic Acidemia (PA) , Methylmalonic Acidemia (MMA)Taiwan
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Baylor College of MedicineCompletedUrea Cycle Disorders | Argininosuccinic Aciduria | Argininosuccinate Lyase DeficiencyUnited States
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National Human Genome Research Institute (NHGRI)TerminatedCitrullinemia | Argininosuccinic Aciduria | Hyperargininemia | Ornithine Carbamoyltransferase Deficiency | Carbamoyl-Phosphate Synthase I Deficiency | N-Acetylglutamate Synthase DeficiencyUnited States
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Oregon Health and Science UniversityTerminatedImmune System Diseases | Lipid Metabolism, Inborn Errors | Mevalonic Aciduria | Mevalonate Kinase Deficiency | Periodic Fever Syndromes, HereditaryUnited States
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King Abdullah International Medical Research CenterCompletedPropionic Acidemia | Methylmalonic AcidemiaSaudi Arabia
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Universität des SaarlandesInstitute of Alternative and Sustainable Nutrition (IFANE)CompletedCobalamin DeficiencyGermany
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Brendan LeeEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsCompletedAmino Acid Metabolism, Inborn Errors | Urea Cycle Disorders | Argininosuccinic AciduriaUnited States
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Wellstat TherapeuticsCompletedHereditary Orotic AciduriaUnited States
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Mendel TuchmanUniversity of Colorado, Denver; Children's Hospital of Philadelphia; University... and other collaboratorsTerminatedPropionic Acidemia | Methylmalonic AcidemiaUnited States
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Haukeland University HospitalNorwegian Foundation for Health and Rehabilitation; Foundation to Promote Research...CompletedVitamin B 12 DeficiencyNorway
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USDA, Western Human Nutrition Research CenterCompleted
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Children's Hospital of PhiladelphiaEunice Kennedy Shriver National Institute of Child Health and Human Development...WithdrawnUrea Cycle Disorders, Inborn | Inborn Errors of Metabolism | Propionic Acidemia | Methylmalonic Acidemia | Carbamyl Phosphate Synthetase Deficiency
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Northwell HealthCompletedType 2 Diabetes Mellitus | Vitamin B12 DeficiencyUnited States