Clinical Trials on GM3 Synthase Deficiency
Total 9111 results
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DDC Clinic - Center for Special Needs ChildrenUnknownGM3 Synthase Deficiency
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Polaris GroupCompletedArgininosuccinate Synthetase DeficientUnited States
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Baylor College of MedicineChildren's National Research InstituteRecruitingOrnithine Transcarbamylase Deficiency | Urea Cycle Disorder | Carbamyl Phosphate Synthetase Deficiency | Argininosuccinic Aciduria | Hyperargininemia | Citrullinemia 1 | ARGI Deficiency | ASL Deficiency | ASS Deficiency | NAGS DeficiencyUnited States
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Cytonet GmbH & Co. KGCompletedOrnithine Transcarbamylase Deficiency | Urea Cycle Disorders | Citrullinemia | Carbamoylphosphate Synthetase I DeficiencyGermany
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West Kazakhstan Medical UniversityRecruitingOrnithine Transcarbamylase Deficiency | Biotinidase Deficiency | Citrullinemia | Glutaric Acidemia Type II | Argininosuccinic Aciduria | Maple Syrup Urine Disease | Primary Carnitine Deficiency | Homocystinuria | Carnitine Palmitoyltransferase II Deficiency | Arginase Deficiency | Very Long-chain Acyl-CoA... and other conditionsKazakhstan
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Moscow Regional Research and Clinical Institute...TerminatedGlycogen Synthase Kinase 3Russian Federation
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsTerminatedTay-Sachs Disease | Sandhoff Disease | GM1 Gangliosidoses | GM2 GangliosidosesUnited States
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University Hospital, Strasbourg, FranceUnknownThe Glutathione Synthetase DeficiencyFrance
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Nicholas Ah MewIcahn School of Medicine at Mount Sinai; Boston Children's HospitalRecruitingN-acetylglutamate Synthase (NAGS) DeficiencyUnited States
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The First Hospital of Jilin UniversityNational Natural Science Foundation of ChinaUnknownGenetic Diseases, Inborn | Asparagine Synthetase DeficiencyChina
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SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt)RecruitingGangliosidoses | Galactosialidosis | Sialidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Morquio B Disease | Gm2-Gangliosidosis, Variant B1 | GM2 Activator DeficiencyGermany
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National Human Genome Research Institute (NHGRI)TerminatedCitrullinemia | Argininosuccinic Aciduria | Hyperargininemia | Ornithine Carbamoyltransferase Deficiency | Carbamoyl-Phosphate Synthase I Deficiency | N-Acetylglutamate Synthase DeficiencyUnited States
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Children's Hospital of PhiladelphiaEunice Kennedy Shriver National Institute of Child Health and Human Development...WithdrawnUrea Cycle Disorders, Inborn | Inborn Errors of Metabolism | Propionic Acidemia | Methylmalonic Acidemia | Carbamyl Phosphate Synthetase Deficiency
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Heinrich-Heine University, DuesseldorfCompletedPeripheral Blood Mononuclear Cell | Red Blood Cell | Endothelial NO-synthaseGermany
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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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Azafaros A.G.Active, not recruitingGM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Brazil, France, Germany, Italy, United Kingdom
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University of PittsburghRecruitingLysosomal Storage Diseases | Leukodystrophy | Niemann-Pick Diseases | Batten Disease | Gaucher Disease | Krabbe Disease | Alpha-Mannosidosis | Osteopetrosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | GM1 Gangliosidoses | MPS I | MPS II | Vanishing White Matter Disease | Multiple Sulfatase Deficiency... and other conditionsUnited States
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University of East AngliaCompletedEicosapentaenoic Acid | Cardiovascular Physiological Phenomena | Endothelial Nitric Oxide Synthase | Docosahexaenoic Acid (All-Z Isomer)United Kingdom
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingGM2 Gangliosidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Late Onset Tay-Sachs DiseaseUnited States
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Passage Bio, Inc.Active, not recruitingGM1 Gangliosidosis | GM1 Gangliosidosis, Type I | GM1 Gangliosidosis, Type 2 | Beta-Galactosidase-1 (GLB1) DeficiencyUnited States, Canada, United Kingdom, Turkey, Brazil
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National Institute of Allergy and Infectious Diseases...Terminated
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Mendel TuchmanChildren's Hospital of Philadelphia; University of California, Los Angeles; Icahn... and other collaboratorsCompletedMethylmalonic Acidemia | Carbamoyl-Phosphate Synthase I Deficiency Disease | Propionic Acidemia, Type I and/or Type II | Ornithine Carbamoyltransferase DeficiencyUnited States
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Travere Therapeutics, Inc.Recruiting
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Aeglea BiotherapeuticsTerminatedHomocystinuria Due to Cystathionine Beta-Synthase DeficiencyUnited States, United Kingdom, Australia
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Rigshospitalet, DenmarkUnknownCarbohydrate Metabolism, Inborn Errors | Metabolism, Inborn Errors | Lipid Metabolism, Inborn Errors | Glycogen Storage Disease Type II | Glycogen Storage Disease Type V | VLCAD Deficiency | Glycogen Storage Disease Type III | Phosphoglycerate Kinase Deficiency | Neutral Lipid Storage Disease | Carnitine... and other conditionsDenmark
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Heinrich-Heine University, DuesseldorfCompletedCoronary Artery Disease | Endothelial NO-synthaseGermany
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National Human Genome Research Institute (NHGRI)Sio Gene TherapiesRecruitingGangliosidosis | Lysosomal Diseases | GM1United States
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National Institute of Allergy and Infectious Diseases...Active, not recruitingPGM3 Deficiency | Eosinophilic and/or Atopic Dermatitis | OSMR Deficiency | Primary Localized Cutaneous Amyloidosis | Hereditary Alpha-tryptasemiaUnited States
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Cabaletta BioRecruitingIdiopathic Inflammatory Myopathy | Dermatomyositis | Immune-Mediated Necrotizing Myopathy | Anti-Synthetase SyndromeUnited States
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Baylor College of MedicineCompletedCitrullinemiaUnited States
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Central Hospital, Nancy, FranceUniversity Hospital, Strasbourg, France; CHU de Reims; Centre Hospitalier Universitaire... and other collaboratorsRecruiting
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University Hospital, ToursInstitut National de la Santé Et de la Recherche Médicale, FranceCompletedValidate the French Version of GMS PackageFrance
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Istituto Scientifico Romagnolo per lo Studio e...Pierre Fabre LaboratoriesTerminatedCarcinoma, Non-Small-Cell Lung | Secondary | Advanced Stage IIIB | High Thymidylate Synthase ExpressionItaly
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The Hospital for Sick ChildrenActelionCompletedGangliosidoses GM2Canada
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Shanghai Chest HospitalUnknownNSCLC | SBRT | GM-CSFChina
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Centre Hospitalier Universitaire de LiegeSanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaboratorsRecruitingCongenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditionsBelgium
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University of UlmPfizer, Berlin Germany; Medac, Hamburg, Germany; Study Group Oncology of Gastrointestinal...CompletedColorectal Cancer | Biopsy | Non Resectable Metastasis | Reference Lesion | Thymidylate Synthase QuantitationGermany
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Dr. Anupam SehgalTaysha Gene Therapies, Inc.; GlycoNetActive, not recruitingInfantile GM2 Gangliosidosis (Disorder)Canada
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Children's National Research InstituteActelionCompletedSandhoff Disease | GM2 Gangliosidoses | Tay-SachsUnited States
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Washington University School of MedicineUniversity of California, Davis; Universidad San Francisco de Quito; Pan American...CompletedCholine Deficiency | Vitamin B-12 Deficiency | Lipids Deficiency | Amino Acids DeficiencyEcuador
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Nutrition Institute, SloveniaEuropean Regional Development Fund; Vizera d.o.o.; Frutarom Etol d.o.o.CompletedVitamin B 12 Deficiency | Vitamin d Deficiency | Protein DeficiencySlovenia
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RTI InternationalEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationPrimary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditionsUnited States
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Eli Lilly and CompanyCompletedGrowth Hormone (GH) Deficiency | Short Stature Homeobox Containing Gene (SHOX) Deficiency | SHOX Deficiency-related Disorder | Non-GH-deficient Growth Disorders
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Texas A&M UniversityUnknownCognitive Change | Nutritional Anemia | Diet, Healthy | Nutrient Deficiency | Diet; Deficiency | Visual Spatial Processing | Dietary Deficiency | Dietary B12 Deficiency | Dietary Zinc Deficiency | Dietary Vitamin B12 Deficiency Anemia | Dietary Deficiency of Selenium and Vitamin EUnited States
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National Human Genome Research Institute (NHGRI)RecruitingMetabolic Disease | Purine-Pyrimidine Metabolism | AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2 | UNG, OMIM *191525, Hyper-IgM Syndrome 5 | NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency | UMPS, OMIM *613891, Orotic Aciduria | DHODH, OMIM *126064,... and other conditionsUnited States
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SourseCitruslabsActive, not recruitingMood | Energy Supply; Deficiency | B12 Deficiency VitaminUnited States
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University of California, DavisUniversity of California, San Francisco; University of Rhode Island; Ethiopian... and other collaboratorsEnrolling by invitationVitamin B 12 Deficiency | Folate Deficiency | Iodine Deficiency | Anemia Deficiency | Salt Intake | Anemia MacrocyticEthiopia
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ProofPilotJoovvUnknownTestosterone Deficiency | Estrogen DeficiencyUnited States
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Meir Medical CenterCompletedFolic Acid Deficiency | Vitamin B12 DeficiencyIsrael
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University Medical Centre LjubljanaCompletedBody Weight | IVF | Nutrient Deficiency | Dietary Vitamin B12 Deficiency Anemia | Dietary Folate Deficiency AnemiaSlovenia