Clinical Trials on Lipid Metabolism, Inborn Errors in United States
Total 3369 results
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ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseUnited States, Netherlands, Hungary, United Kingdom, Czechia, Norway, Slovenia, Spain, Finland, France, Italy, Switzerland
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Massachusetts General HospitalRecruiting
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Chiesi Farmaceutici S.p.A.Active, not recruitingFabry DiseaseUnited States, Netherlands, Australia, Hungary, United Kingdom, Canada, Czechia, Finland, France, Italy, Norway, Slovenia, Spain
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University of California, San DiegoCompleted
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Genzyme, a Sanofi CompanyCompletedGaucher Disease, Type 1 | Cerebroside Lipidosis Syndrome | Glucocerebrosidase Deficiency Disease | Glucosylceramide Beta-Glucosidase Deficiency Disease | Gaucher Disease, Non-Neuronopathic FormUnited States, Israel, Mexico, Russian Federation, Argentina, Italy
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PTC TherapeuticsRecruitingPhenylketonuriaUnited States
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Boston Children's HospitalBioMarin PharmaceuticalCompletedPhenylketonuriasUnited States
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University of NebraskaCompleted
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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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Oregon Health and Science UniversityTerminated
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Sheffield Teaching Hospitals NHS Foundation TrustCompleted
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Idorsia Pharmaceuticals Ltd.CompletedGM2 Gangliosidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Gaucher Disease, Type 2 | AB Variant Gangliosidosis GM2United States, Spain, Germany, Italy, Belgium, Brazil, France, Portugal, Switzerland, United Kingdom
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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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Washington University School of MedicineUniversity of Missouri-Columbia; BioMarin PharmaceuticalCompleted
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Shoji YanoBioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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Vitaflo International, LtdOregon Health and Science UniversityCompleted
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Children's Hospital Medical Center, CincinnatiBioMarin PharmaceuticalCompleted
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University of MiamiCompletedPhenylketonuriaUnited States
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Children's Hospital of PhiladelphiaCompleted
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Eunice Kennedy Shriver National Institute of Child...Completed
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Wake Forest University Health SciencesCompletedCardiovascular Diseases | Diabetes | Metabolic Syndrome | Liver Fat | Cardiovascular Risk Factor | Diet Habit | Dietary Modification | High Cholesterol | Triglycerides High | Low-density-lipoprotein-type | HDL Cholesterol, Low Serum | High Blood Sugar | Intra-abdominal Fat | High Density Lipoprotein DeficiencyUnited States
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National Human Genome Research Institute (NHGRI)RecruitingPropionic Acidemia | Metabolic Disease | Organic AcidemiaUnited States
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Boston Children's HospitalCompletedSmith-Lemli-Opitz SyndromeUnited States
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Forbes Porter, M.D.Eunice Kennedy Shriver National Institute of Child Health and Human Development...Completed
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Vitaflo International, LtdCompleted
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PTC TherapeuticsAxio Research. LLCCompleted
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Rocket Pharmaceuticals Inc.Active, not recruiting
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University of Southern CaliforniaNational PKU AllianceActive, not recruitingPhenylketonuriasUnited States
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Emory UniversityBioMarin Pharmaceutical; Atlanta Clinical and Translational Science InstituteCompletedPhenylketonuriaUnited States
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National Human Genome Research Institute (NHGRI)Recruiting
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Vitaflo International, LtdBirmingham Women's and Children's NHS Foundation TrustCompletedPhenylketonuria | Inborn Errors of MetabolismUnited Kingdom
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CENTOGENE GmbH RostockCompletedPyruvate Kinase DeficiencyUnited States
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University of South FloridaNational Institutes of Health (NIH)CompletedUrea Cycle DisordersUnited States
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PTC TherapeuticsCompletedLeigh SyndromeUnited States
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Alexion Pharmaceuticals, Inc.RecruitingHypophosphatasiaUnited States, Japan, Germany, Turkey, Italy, India, Australia, France, United Kingdom, Argentina, Canada
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Applied Therapeutics, Inc.Active, not recruiting
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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Emory UniversityNational PKU AllianceCompleted
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Oregon Health and Science UniversityTerminatedSmith-Lemli-Opitz SyndromeUnited States
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National Human Genome Research Institute (NHGRI)RecruitingArterial Calcification Due to Deficiency of CD73United States
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HemoShear TherapeuticsPrometrika, LLC; AllStripes Research Inc.; Genome MedicalTerminatedPropionic Acidemia | Methylmalonic AcidemiaUnited States, Canada, United Kingdom
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Ultragenyx Pharmaceutical IncCompletedGSD1Netherlands, United States, Canada, Spain
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University of CambridgeLund University; GE Healthcare; Cambridge University Hospitals NHS Foundation... and other collaboratorsRecruitingAtherosclerosis | Carotid Artery Plaque | Hypercholesterolemia | Hypercholesterolemia, FamilialUnited Kingdom
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, Canada, United Arab Emirates, United Kingdom
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University of Missouri-ColumbiaBioMarin PharmaceuticalEnrolling by invitationPhenylketonuriasUnited States