Search clinical trials for: Carbohydrate Metabolism, Inborn
Total 15191 results
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Alnylam PharmaceuticalsApproved for marketingPrimary HyperoxaluriaBelgium
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Vitaflo International, LtdUniversity College London Hospitals; University Medical Center Groningen; University... and other collaboratorsCompletedGlycogen Storage DiseaseUnited States, United Kingdom, France, Netherlands
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SanofiCompletedPompe Disease | Mucopolysaccharidosis Type I (MPS I)Italy
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University of ManchesterUniversity College, London; Manchester University NHS Foundation Trust; Orchard... and other collaboratorsActive, not recruitingMucopolysaccharidosis Type IIIAUnited Kingdom
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Rocket Pharmaceuticals Inc.Active, not recruiting
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Mayo ClinicChildren's Hospital of Philadelphia; Seattle Children's HospitalTerminated
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Maastricht University Medical CenterPfizerCompleted
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Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyEnrolling by invitationKidney Diseases | Urologic Diseases | Genetic Disease | Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 2 (PH2) | Primary Hyperoxaluria Type 3 (PH3)United States, France, Germany, Japan, Lebanon, Spain, United Kingdom, Australia, Canada, Italy, Netherlands, Norway, Turkey
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SanofiCompletedOsteochondrodysplasiaUnited States
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JCR Pharmaceuticals Co., Ltd.Enrolling by invitationMucopolysaccharidosis IIUnited States
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LIB Therapeutics LLCMedpace, Inc.CompletedHeterozygous Familial HypercholesterolemiaUnited States, Israel, Norway, South Africa, Turkey
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REGENXBIO Inc.Active, not recruitingHurler Syndrome | Hurler-Scheie Syndrome | Mucopolysaccharidosis Type I (MPS I)United States, Brazil, Israel
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University Children's Hospital, ZurichSanofiCompleted
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PTC TherapeuticsCompletedLeigh SyndromeUnited States
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Rocket Pharmaceuticals Inc.Completed
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Purdue UniversityCompletedLactose Intolerance, Adult TypeUnited States
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REGENXBIO Inc.Active, not recruitingMucopolysaccharidosis Type II (MPS II)United States, Brazil
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REGENXBIO Inc.Active, not recruitingMucopolysaccharidosis Type II (MPS II)United States, Canada
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Spark TherapeuticsActive, not recruitingLysosomal Storage Diseases | Glycogen Storage Disease Type II | Glycogen Storage Disease Type 2 | Pompe Disease (Late-onset) | Pompe Disease | LOPD | Acid Maltase DeficiencyUnited States, Canada, Netherlands, France, Denmark, Germany, Italy, United Kingdom
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agnes chenUniversity of California, Los Angeles; National Institute of Neurological Disorders... and other collaboratorsTerminatedMucopolysaccharidosis I | Cognitive DeclineUnited States
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Duke UniversityGenzyme, a Sanofi CompanyActive, not recruitingPompe Disease (Late-onset) | Pompe Disease | GAA DeficiencyUnited States
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REGENXBIO Inc.Enrolling by invitation
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Dr. Prayuth SudathipMedicines for Malaria VentureCompletedMalaria, Vivax | G6PD DeficiencyThailand
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Agios Pharmaceuticals, Inc.CompletedPyruvate Kinase DeficiencyUnited States, Italy, Netherlands
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Ultragenyx Pharmaceutical IncTerminatedGlycogen Storage Disease Type IIIUnited States, Netherlands
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Genzyme, a Sanofi CompanyCompleted
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Genzyme, a Sanofi CompanyActive, not recruitingGlycogen Storage Disease Type IIFrance
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Hospital de Clinicas de Porto AlegreConselho Nacional de Desenvolvimento Científico e Tecnológico; Fundação Médica...CompletedGlycogen Storage Disease Type IBrazil
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ShireCompleted
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University of FloridaLive Like Bella Pediatric Cancer ResearchCompletedCommon Variable Immunodeficiency | Severe Combined Immunodeficiency | Sickle Cell Disease | Hurler Syndrome | Chronic Granulomatous Disease | Thalassemia | Wiskott-Aldrich Syndrome | Adrenoleukodystrophy | X-linked Lymphoproliferative Disease | Diamond Blackfan Anemia | Bone Marrow Failure Syndrome | Hemophagocytic... and other conditionsUnited States
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Greenwood Genetic CenterCompletedGalactosialidosis | Sialidosis | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Beta Mannosidosis | Mucolipidosis II | Mucolipidosis III | Schindler DiseaseUnited States
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Amicus TherapeuticsCompletedPompe Disease (Late-onset)United States, Australia, Korea, Republic of, Japan, Taiwan, Belgium, Slovenia, New Zealand, United Kingdom, France, Argentina, Canada, Spain, Bosnia and Herzegovina, Germany, Sweden, Hungary, Austria, Bulgaria, Denmark, Greece, I... and more
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Agios Pharmaceuticals, Inc.TerminatedAnemia | Anemia, Hemolytic | Pyruvate Kinase Deficiency | Pyruvate Kinase Deficiency AnemiaUnited States, United Kingdom, Italy, Spain, Netherlands
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Manchester University NHS Foundation TrustCompletedMucopolysaccharidosis Type IIUnited Kingdom
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Homology Medicines, IncWithdrawnMucopolysaccharidosis IIUnited States, Canada
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Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyAvailablePrimary Hyperoxaluria Type 1 (PH1)
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Allievex CorporationEnrolling by invitationMPS III BUnited States, Germany, Turkey, United Kingdom, Colombia
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University of FloridaEunice Kennedy Shriver National Institute of Child Health and Human Development...Completed
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Chiesi Farmaceutici S.p.A.Completed
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Chiesi Farmaceutici S.p.A.CompletedAlpha-MannosidosisFrance
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Amicus TherapeuticsAvailablePompe Disease Infantile-OnsetTaiwan, United States, Italy
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Amicus TherapeuticsAvailable
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Ultragenyx Pharmaceutical IncAbeona Therapeutics, IncTerminatedMucopolysaccharidosis III | Sanfilippo Syndrome | MPS IIIA | Sanfilippo AUnited States, Spain, Australia
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Arizona State UniversityWashington State UniversityEnrolling by invitationLanguage Disorders in Children | Classic Galactosemia | Speech Disorders in ChildrenUnited States
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TakedaTakeda Development Center Americas, Inc.Active, not recruitingHunter SyndromeUnited States, Germany, Malaysia, Philippines, Thailand, Dominican Republic, Serbia, Vietnam
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Genzyme, a Sanofi CompanyActive, not recruitingGlycogen Storage Disease Type II-Pompe's DiseaseUnited States, France, Japan, Taiwan, United Kingdom
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University of FloridaEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsActive, not recruitingPyruvate Dehydrogenase Complex DeficiencyUnited States
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Asklepios Biopharmaceutical, Inc.Active, not recruiting