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Clinical Trials on Pyruvate Metabolism, Inborn Errors in United States
Total 2366 results
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Forge Biologics, IncNot yet recruitingKrabbe DiseaseUnited States
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Horizon Therapeutics Ireland DACCompletedUncontrolled Gout | Kidney TransplantUnited States
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Ultragenyx Pharmaceutical IncAbeona Therapeutics, IncEnrolling by invitationMucopolysaccharidosis III-AUnited States, Australia, Spain
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Travere Therapeutics, Inc.Active, not recruiting
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States, Canada
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BioMarin PharmaceuticalApproved for marketing
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PfizerCompletedHeterozygous Familial HypercholesterolemiaCanada, Netherlands, United Kingdom, United States, Spain, Poland, Finland, Norway, South Africa, Italy, Bulgaria
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Masonic Cancer Center, University of MinnesotaRecruitingMucopolysaccharidosis Type I | Hematopoietic Cell TransplantationUnited States
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Masonic Cancer Center, University of MinnesotaBioMarin PharmaceuticalTerminated
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Masonic Cancer Center, University of MinnesotaCompletedMucopolysaccharidosis I | Hurler SyndromeUnited States
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Regeneron PharmaceuticalsCompletedHomozygous Familial HypercholesterolemiaUnited States, Netherlands, South Africa, Australia, Austria, Canada, Czechia, France, Greece, Italy, Japan, Ukraine
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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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Kyowa Hakko Kirin Pharma, Inc.Completed
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AmgenCompletedHeterozygous Familial HypercholesterolemiaUnited States, Canada, Italy, Norway, Belgium, Turkey, Australia, New Zealand, Austria, Czechia, South Africa, Poland, Spain, Switzerland, Netherlands, Taiwan, Finland, Brazil, Portugal, Slovenia, Russian Federation, Hungary, United... and more
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Amicus TherapeuticsTerminatedBatten Disease | Neuronal Ceroid Lipofuscinosis CLN6 | Neuronal Ceroid Lipofuscinosis CLN3United States
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Kastle Therapeutics, LLCCompletedHypercholesterolemia | Heterozygous FamilialUnited States, Italy, Poland, Ukraine, United Kingdom, Germany, Hong Kong, Taiwan, Spain, Argentina, Belgium, Israel, Malaysia, South Africa, Turkey, Netherlands, Korea, Republic of, Canada, New Zealand, Russian Federation, Brazil, Austral... and more
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Zymenex A/SEuropean CommissionCompletedA Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-MannosidosisAlpha-MannosidosisDenmark, United Kingdom, France, Germany, Poland
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Lundquist Institute for Biomedical Innovation at...Not yet recruitingMucopolysaccharidosis III | Sanfilippo SyndromeUnited States
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Sanguine BiosciencesDenali Therapeutics Inc.RecruitingMucopolysaccharidosis III-AUnited States
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Maggie's Pearl, LLCActive, not recruitingPmm2-CDG | Phosphomannomutase 2 Deficiency | Phosphomannomutase 2 Congenital Disorder of Glycosylation | Phosphomannomutase II Congenital Disorder of Glycosylation | Phosphomannomutase II DeficiencyUnited States
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Masonic Cancer Center, University of MinnesotaCompleted
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingGaucher Disease Type 1United States
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Children's National Research InstituteActelionCompletedSandhoff Disease | GM2 Gangliosidoses | Tay-SachsUnited States
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NovartisCompletedArthritis, GoutyUnited States, Switzerland, United Kingdom
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InventisBio Co., LtdRecruiting
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Weill Medical College of Cornell UniversityNational Institute of Neurological Disorders and Stroke (NINDS); National Institutes... and other collaboratorsCompletedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States
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Eunice Kennedy Shriver National Institute of Child...CompletedInfantile Neronal Ceroid LipofuscinosisUnited States
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Weill Medical College of Cornell UniversityTerminatedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States
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Urica Therapeutics Inc.RecruitingHyperuricemia | GoutUnited States
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Talaris Therapeutics Inc.Duke UniversityNo longer availableMetachromatic LeukodystrophyUnited States
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ShireCompletedGaucher Disease, Type 1Spain, Israel, United States, India, Paraguay, United Kingdom, Russian Federation, Tunisia, Argentina
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Alexion PharmaceuticalsCompletedMucopolysaccharidosis IIIBUnited States, United Kingdom, Spain
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National Human Genome Research Institute (NHGRI)Recruiting
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St. Jude Children's Research HospitalNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Assisi...CompletedGalactosialidosisUnited States
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National Eye Institute (NEI)CompletedCystinosisUnited States
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AVROBIOTerminatedGaucher DiseaseUnited States, Canada
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BioMarin PharmaceuticalCompletedTetrahydrobiopterin Deficiencies | Hyperphenylalaninemia, Non-PhenylketonuricGermany, United States
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Inozyme PharmaEngage Health Inc.; GACI GlobalCompletedGeneralized Arterial Calcification in Infancy | Autosomal Recessive Hypophosphatemic Rickets Type 2United States
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University of GlasgowCompletedPhenylketonuria (PKU)United Kingdom
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Cycle Pharmaceuticals Ltd.ParexelCompletedHereditary Tyrosinemia, Type ISouth Africa
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PTC TherapeuticsCompletedPhenylketonuriaSpain, United States, Australia, Germany, United Kingdom, Brazil, Denmark, Canada, France, Georgia, Italy, Mexico, Netherlands, Portugal, Turkey
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Amryt PharmaRecruitingHomozygous Familial HypercholesterolemiaItaly, United States, Argentina, Canada, France, Greece, Netherlands, United Kingdom
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Stanford UniversityRecruiting
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University of California, San DiegoEnrolling by invitationCystinosisUnited States
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Duke UniversityCompleted
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Mark BatshawEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsTerminatedUrea Cycle DisordersUnited States, Canada
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BioMarin PharmaceuticalTerminatedLate-onset Pompe DiseaseUnited States, United Kingdom
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University College, LondonRecruitingOrnithine Transcarbamylase DeficiencyUnited Kingdom
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University of ReadingCompletedMilk IntoleranceUnited Kingdom
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Cycle Pharmaceuticals Ltd.ParexelCompletedHereditary Tyrosinemia, Type ISouth Africa