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Clinical Trials on Pyruvate Metabolism, Inborn Errors in United States
Total 2358 results
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jasvinder singhNovartis; Takeda; Regeneron Pharmaceuticals; Birmingham, Alabama VA Medical CenterWithdrawn
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University of South FloridaCompleted
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Alexion Pharmaceuticals, Inc.Not yet recruitingHypophosphatasiaUnited States, Taiwan, Thailand, Vietnam, United Kingdom, Korea, Republic of, Israel, Italy, Australia, Turkey, France, Poland, Germany, Spain, Belgium, China, Canada, Brazil, Argentina, Mexico
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Ardea Biosciences, Inc.Completed
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Lysosomal and Rare Disorders Research and Treatment...UnknownGaucher DiseaseUnited States
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Geisinger ClinicCompletedHypercholesterolemia | Hypercholesterolemia, Familial | Hypercholesteremia in Children | Hyperlipidemia in ChildrenUnited States
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University of Texas Southwestern Medical CenterWithdrawnFamilial HypercholesterolemiaUnited States
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Vanderbilt University Medical CenterTerminatedAlbright Hereditary Osteodystrophy | Pseudohypoparathyroidism and Pseudopseudohypoparathyroidism | Pseudohypoparathyroidism Type IaUnited States
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Homology Medicines, IncEnrolling by invitationPhenylketonuria | PAH DeficiencyUnited States
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Aegerion Pharmaceuticals, Inc.CompletedFamilial HypercholesterolemiaUnited States, Canada, Italy, South Africa
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BioMarin PharmaceuticalActive, not recruiting
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SynlogicCompletedHealthy | PhenylketonuriaUnited States
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Applied Therapeutics, Inc.CompletedClassic GalactosemiaUnited States
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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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University of MinnesotaRecruitingGaucher DiseaseUnited States
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TakedaCompletedGaucher DiseaseUnited States
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Genzyme, a Sanofi CompanyCompleted
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Brigham and Women's HospitalGenzyme, a Sanofi CompanyUnknown
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Purdue UniversityRecruitingLactose IntoleranceUnited States
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Mayo ClinicNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Orphan...CompletedHyperoxaluriaUnited States
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University of Massachusetts, WorcesterTakedaCompleted
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Rutgers, The State University of New JerseyCompleted
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, France, United Kingdom, Canada, Netherlands, Russian Federation, Turkey, Australia
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Connecticut Children's Medical CenterJohns Hopkins University; Eunice Kennedy Shriver National Institute of Child... and other collaboratorsRecruitingAlbright Hereditary Osteodystrophy | Pseudopseudohypoparathyroidism | Pseudohypoparathyroidism Type 1AUnited States
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ModernaTX, Inc.RecruitingPropionic AcidemiaUnited States, Spain, Netherlands, France, United Kingdom, Australia, Canada, Italy
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Mirum Pharmaceuticals, Inc.Completed
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Regeneron PharmaceuticalsCompletedHomozygous Familial HypercholesterolemiaUnited States, Netherlands, Canada
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PTC TherapeuticsGenzyme, a Sanofi CompanyTerminatedAmino Acid Metabolism, Inborn ErrorsFrance, United Kingdom, Italy, Belgium, Germany, Switzerland
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University of Missouri-ColumbiaCompleted
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Indiana UniversityCompletedChronic Kidney DiseaseUnited States
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Oregon Health and Science UniversityUniversity of Oregon; Oregon State UniversityRecruitingCarbohydrate IntoleranceUnited States
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Eva Morava-KoziczNot yet recruitingPGM1-CDG - Phosphoglucomutase 1-Related Congenital Disorder of GlycosylationUnited States
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OxTheraCompletedPrimary HyperoxaluriaUnited States
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BioMarin PharmaceuticalCompleted
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Amicus TherapeuticsActive, not recruiting
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Massachusetts General HospitalAlexion PharmaceuticalsUnknownCholesterol Ester Storage Disease | Lysosomal Acid Lipase DeficiencyUnited States
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Kastle Therapeutics, LLCIonis Pharmaceuticals, Inc.CompletedHypercholesterolemia, FamilialUnited States, Netherlands
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TakedaCompleted
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Origin BiosciencesCompletedMolybdenum Cofactor Deficiency, Type AUnited States, Spain, Israel, Norway, Turkey, United Kingdom
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Ritter Pharmaceuticals, Inc.CompletedLactose IntoleranceUnited States
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Duke UniversityRecruiting
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Duke UniversityRecruitingAdult Polyglucosan Body Disease | Glycogen Storage Disease Type IV | GSD4 | GSD IV | APBDUnited States
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Cerenis Therapeutics, SACompletedHomozygous Familial HypercholesterolemiaUnited States, Canada, Italy, Netherlands, United Kingdom
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Manchester University NHS Foundation TrustFDNA Inc.CompletedInborn Errors of Metabolism | Dysmorphic Features | Multiple AnomaliesUnited Kingdom
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Horizon Pharma Ireland, Ltd., Dublin IrelandCompletedUrea Cycle DisordersUnited States
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University of PennsylvaniaUniversity of Amsterdam; University of Cape Town; University of Witwatersrand...RecruitingHomozygous Familial HypercholesterolemiaUnited States, Netherlands, South Africa
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Regeneron PharmaceuticalsSanofiCompletedHypercholesterolemiaFrance, United States
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Alexion Pharmaceuticals, Inc.RecruitingHypophosphatasiaItaly, Spain, Israel, Korea, Republic of, Belgium, Germany, United States, China, France, Taiwan, Austria, Japan, India, Australia, Argentina, Poland, Turkey, Brazil, Thailand, United Kingdom, Canada, Slovakia, Czechia