Search clinical trials for: Carbohydrate Metabolism, Inborn Errors
Total 17006 results
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NYU Langone HealthNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)CompletedCystinuria | Primary Hyperoxaluria | Dent Disease | Adenine Phosphoribosyl Transferase DeficiencyUnited States, Iceland
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Pompe DiseaseUnited States
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LMU KlinikumCompletedHealthy | Myotonic Dystrophy Type 2 | Pompe Disease (Late-onset) | Spinal Muscular Atrophy Type 3 | Myotonic Dystrophy Type 1 (DM1) | Facioscapulohumeral Muscular Dystrophy 1 | Inclusion Body Myositis, SporadicGermany
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Duke UniversityWithdrawnPompe Disease (Late-onset)United States
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Applied Therapeutics, Inc.Active, not recruiting
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CENTOGENE GmbH RostockTerminatedDeafness | Immunodeficiency | Mental Retardation | Gingival Hypertrophy | Skeletal Abnormalities | Facial DysmorphismGermany, Switzerland
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Abeona Therapeutics, IncTerminatedMucopolysaccharidosis III-BUnited States, Germany, France
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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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Taysha Gene Therapies, Inc.Withdrawn
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PATHFred Hutchinson Cancer CenterCompletedG6PD DeficiencyUnited States
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Instituto de Investigación Hospital Universitario...Go Fruselva, S.L.Completed
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TakedaCompletedMucopolysaccharidosis (MPS)Brazil
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Abeona Therapeutics, IncTerminatedMucopolysaccharidosis Type 3 BUnited States, Germany, France, Spain
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REGENXBIO Inc.CompletedMucopolysaccharidosis IIBrazil, United States, United Kingdom
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type IIChina
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Genzyme, a Sanofi CompanyTerminatedGlycogen Storage Disease Type II (GSD-II) | Pompe Disease | Glycogenesis 2 Acid Maltase DeficiencyUnited States, Israel
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University of FloridaLacerta TherapeuticsCompletedPompe DiseaseUnited States
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Genzyme, a Sanofi CompanyCompletedPompe Disease | Glycogen Storage Disease Type II (GSD II)United States, Russian Federation, United Kingdom, Bulgaria, India, Ukraine
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Swedish Orphan BiovitrumCompletedSanfilippo Syndrome Type A (MPS IIIA)United States, Turkey
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University Medical Center GroningenUltragenyx Pharmaceutical IncCompletedGlycogen Storage Disease Type IANetherlands
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Aadi Bioscience, Inc.Withdrawn
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Reneo Pharma LtdCompletedMcArdle DiseaseSpain, United Kingdom
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OxTheraFP7-SME-2013 Research for the benefit of SMEs programCompletedPrimary HyperoxaluriaGermany
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Agios Pharmaceuticals, Inc.CompletedAnemia, Hemolytic | Pyruvate Kinase DeficiencyUnited States, United Kingdom, Netherlands, Canada, Denmark, France, Italy, Ireland, Thailand
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University Hospital, ToursCompletedMucopolysaccharidosesFrance
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Areeg El-GharbawyUltragenyx Pharmaceutical IncCompletedGlycogen Storage Disease Type IUnited States
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OxTheraCompletedPrimary HyperoxaluriaFrance, United Kingdom, Spain, Belgium, United States, Germany, Tunisia
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Jerry Vockley, MD, PhDUltragenyx Pharmaceutical IncNo longer availableBarth Syndrome | Mitochondrial Trifunctional Protein Deficiency | Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency | Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2) | Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency | Glycogen Storage Disorders | Pyruvate Carboxylase Deficiency... and other conditionsUnited States
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OxTheraTerminatedHyperoxaluria, PrimaryUnited Kingdom, Spain, Belgium, United States, Germany, Tunisia
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Fondazione TelethonActive, not recruitingMucopolysaccharidosis Type VIItaly, Turkey
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Swedish Orphan BiovitrumCompletedSanfilippo Syndrome Type A (MPS IIIA)United States, Germany, Turkey
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PATHMahidol Oxford Tropical Medicine Research UnitTerminated
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PATHFundação de Medicina Tropical Dr. Heitor Vieira DouradoCompleted
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PATHCompletedG6PD DeficiencyUnited States
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Duke UniversityCompleted
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Chiesi Farmaceutici S.p.A.CromsourceCompletedAlpha-MannosidosisFrance, Denmark, Austria, Germany, Italy
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Khondrion BVDrug Research Unit Ghent, BelgiumCompletedMitochondrial Disease | MELAS | Leigh Syndrome | LHON | Mitochondrial DNA tRNALeu(UUR) m.3243A<G MutationBelgium
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Fondazione TelethonCompletedPyruvate Dehydrogenase Complex DeficiencyItaly
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Vivante HealthDuke UniversityCompletedLactose IntoleranceUnited States
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Dicerna Pharmaceuticals, Inc.CompletedPrimary Hyperoxaluria Type 3United States, Germany, Netherlands, United Kingdom
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LYSOGENECompletedMucopolysaccharidosis IIIAGermany, Brazil, United Kingdom, France, Netherlands
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LYSOGENEActive, not recruitingMucopolysaccharidosis Type IIIAGermany, United States, United Kingdom, France, Netherlands
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IRCCS Eugenio MedeaUniversity of Pisa; University of MessinaCompleted
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Hasan Kalyoncu UniversityCompleted
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IRCCS San RaffaeleFondazione TelethonActive, not recruiting
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REGENXBIO Inc.Enrolling by invitation
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Duke UniversityCompleted
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BioMarin PharmaceuticalCompletedMorquio A Syndrome | MPS IVA | Mucopolysaccharidosis IV AUnited States, Canada, France, Taiwan, Argentina, Colombia, Spain, Turkey, Japan, Saudi Arabia, Netherlands, Denmark, Korea, Republic of, Brazil, United Kingdom, Germany, Norway, Portugal, Italy, Qatar
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Genzyme, a Sanofi CompanyCompletedPompe DiseaseBelgium, United States, Czechia, Germany, Italy, Taiwan
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Duke UniversityGenzyme, a Sanofi CompanyCompletedNeuropathy | Myopathy | Glycogen Storage Disease Type II (Late-onset Pompe Disease)United States