Clinical Trials on Steroid Metabolism, Inborn Errors
Total 4359 results
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Vitaflo International, LtdBirmingham Women's and Children's NHS Foundation TrustRecruiting
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Jnana TherapeuticsRecruitingPhenylketonuriaUnited States, Australia
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University Medical Centre LjubljanaNot yet recruitingPhenylketonurias | Nutritional and Metabolic Diseases
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Applied Therapeutics, Inc.CompletedClassic GalactosemiaUnited States
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SynlogicTerminatedPhenylketonuriaUnited States, Canada, Georgia, Turkey
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University of Southern CaliforniaNational PKU AllianceActive, not recruitingPhenylketonuriasUnited States
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Boston Children's HospitalBioMarin PharmaceuticalCompletedPhenylketonuriasUnited States
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University of NebraskaCompleted
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Homology Medicines, IncTerminated
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University of Missouri-ColumbiaBioMarin PharmaceuticalEnrolling by invitationPhenylketonuriasUnited States
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APR Applied Pharma Research s.a.Terminated
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University Hospital Inselspital, BerneUniversity of ZurichCompleted
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Applied Therapeutics, Inc.Active, not recruitingClassic GalactosemiaUnited States
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University Hospitals Cleveland Medical CenterBioMarin PharmaceuticalCompleted
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The University of Texas Medical Branch, GalvestonTerminated
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Boston Children's HospitalBioMarin PharmaceuticalCompleted
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NestléWithdrawn
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Universidade do PortoBioMarin Pharmaceutical; Centro Hospitalar do PortoCompleted
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University of British ColumbiaVitaflo International, LtdUnknown
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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Nutricia UK LtdWithdrawn
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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Academisch Ziekenhuis MaastrichtCompleted
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University of Wisconsin, MadisonBoston Children's HospitalCompletedPhenylketonuriaUnited States
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BioMarin PharmaceuticalTerminatedPhenylketonuriaItaly, Switzerland
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Emory UniversityBioMarin Pharmaceutical; Atlanta Clinical and Translational Science InstituteCompletedPhenylketonuriaUnited States
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Children's National Research InstituteGeorgetown UniversityWithdrawn
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Oregon Health and Science UniversityTerminated
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Merck KGaA, Darmstadt, GermanySmerud Medical Research International AS; Merck Serono NorwayCompleted
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BioMarin PharmaceuticalCompletedStudy of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006PhenylketonuriaUnited States
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The Kennedy Institute-National Eye ClinicSygekassernes HelsefondCompleted
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University Children's Hospital, ZurichRecruitingUrea Cycle DisordersSwitzerland
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Vanderbilt University Medical CenterRecruitingPseudohypoparathyroidism Type 1a | Albright Hereditary Osteodystrophy | PseudohypoparathyroidismUnited States
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Ashley ShoemakerEnrolling by invitationPseudohypoparathyroidism Type 1a | Albright Hereditary Osteodystrophy | PseudohypoparathyroidismUnited States
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Vanderbilt University Medical CenterMassachusetts General HospitalCompletedPseudohypoparathyroidism Type 1a | Albright Hereditary OsteodystrophyUnited States
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Ultragenyx Pharmaceutical IncTerminatedGlycogen Storage Disease Type IIIUnited States, Spain, Italy
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Chiesi Farmaceutici S.p.A.Completed
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Zymenex A/SCompleted
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Eunice Kennedy Shriver National Institute of Child...Completed
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Zymenex A/SEuropean CommissionCompletedAlpha-MannosidosisBelgium, Denmark, Spain, United Kingdom
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Brain-Gut Research GroupRecruitingFructose Intolerance | Functional Gastrointestinal Disorders | Lactose IntoleranceSwitzerland
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Agios Pharmaceuticals, Inc.CompletedPyruvate Kinase DeficiencyUnited States, Italy, Netherlands
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CENTOGENE GmbH RostockCompletedPyruvate Kinase DeficiencyUnited States
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Agios Pharmaceuticals, Inc.CompletedAnemia, Hemolytic | Pyruvate Kinase DeficiencyUnited States, Spain, Switzerland, Turkey, Germany, France, United Kingdom, Japan, Netherlands, Brazil, Canada, Czechia, Denmark, Italy, Korea, Republic of, Thailand
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Icahn School of Medicine at Mount SinaiMayo Clinic; Baylor College of Medicine; Children's Hospital of Philadelphia; University... and other collaboratorsRecruitingCongenital Disorders of GlycosylationUnited States
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HemoShear TherapeuticsPrometrika, LLC; AllStripes Research Inc.; Genome MedicalTerminatedPropionic Acidemia | Methylmalonic AcidemiaUnited States, Canada, United Kingdom
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Rigshospitalet, DenmarkUniversity College, LondonCompletedMcArdle DiseaseDenmark, United Kingdom