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Clinical Trials on Beta-galactosidase-1 Deficiency
Total 361 results
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HaEmek Medical Center, IsraelUnknownIron Deficiency | ThalassemiaIsrael
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Ultragenyx Pharmaceutical IncAvailableGlucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
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IpsenCompletedGrowth Disorders | Insulin-Like Growth Factor-1 DeficiencyUnited States
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Applied Genetic Technologies CorpNational Heart, Lung, and Blood Institute (NHLBI)CompletedAlpha-1 Antitrypsin DeficiencyUnited States, Ireland
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University of FloridaRecruitingAlpha-1 Antitrypsin Deficiency | Cystic Fibrosis (CF) | AATD | AAT DeficiencyUnited States
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Arrowhead PharmaceuticalsActive, not recruitingAlpha 1-Antitrypsin DeficiencyAustria, Germany, United Kingdom
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Dicerna Pharmaceuticals, Inc.WithdrawnAlpha 1-Antitrypsin DeficiencyNew Zealand
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Mereo BioPharmaSyneos HealthCompletedEmphysema | Alpha 1-Antitrypsin Deficiency | COPDUnited Kingdom, Belgium, United States, Denmark, Canada, Sweden, Poland, Spain
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Ultragenyx Pharmaceutical IncCompletedGlucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)United States, Spain, Australia, France, Israel, Italy, United Kingdom
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University of Massachusetts, WorcesterNational Heart, Lung, and Blood Institute (NHLBI); University of Florida; National... and other collaboratorsCompletedAlpha 1-Antitrypsin DeficiencyUnited States
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Genzyme, a Sanofi CompanyRecruitingGaucher Disease | Cerebroside Lipidosis Syndrome | Glucocerebrosidase Deficiency Disease | Glucosylceramide Beta-Glucosidase Deficiency DiseaseHungary, Croatia, United States, Australia, Belgium, Brazil, Bulgaria, Canada, Denmark, Germany, Greece, Hong Kong, India, Indonesia, Italy, Jordan, Korea, Republic of, Kuwait, Lebanon, Malaysia, Pakistan, Philippines, Poland, Saudi Arabia and more
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Juan PascualActive, not recruitingGlucose Transporter Type 1 Deficiency Syndrome | GLUT1DS1United States
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Janssen Sciences Ireland UCCompletedImmunodeficiency Virus Type 1, HumanUnited States, France, Poland, United Kingdom, Belgium, Germany, Spain, Canada, Russian Federation, Puerto Rico
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CSL BehringCompletedEmphysema | Alpha 1-proteinase Inhibitor DeficiencyAustralia, Canada, Czech Republic, Denmark, Estonia, Finland, Germany, Ireland, Poland, Romania, Sweden
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Adverum Biotechnologies, Inc.CompletedAlpha 1-Antitrypsin DeficiencyUnited States
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Inhibrx, Inc.CompletedAlpha-1 Antitrypsin Deficiency | AATDUnited States, New Zealand, United Kingdom
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Coram Clinical TrialsCompletedAlpha-1 Anti-trypsin DeficiencyUnited States
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University of Alabama at BirminghamNational Institutes of Health (NIH); Mereo BioPharmaCompletedEmphysema or COPD | Alpha-1 Antitrypsin Deficiency (AATD) | Pi*ZZ, Pi*SZ, Pi*Null, Another Rare Phenotype/Genotype Known to be Associated With Either Low or Functionally Impaired AAT Including F or I MutationsUnited States
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University of the PunjabThe National HIV/AIDS ProgrammeCompletedHIV-1-infection | Hypovitaminosis DPakistan
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Baxalta now part of ShireBaxalta Innovations GmbH, now part of ShireTerminatedChronic Obstructive Pulmonary Disease | Alpha1-antitrypsin DeficiencyUnited States, Canada, Australia
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ShireWithdrawnChronic Obstructive Pulmonary Disease | Alpha1-antitrypsin Deficiency
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ANRS, Emerging Infectious DiseasesEuropean and Developing Countries Clinical Trials Partnership (EDCTP); European...RecruitingTuberculosis | HIV-1-infection | Immuno-DeficiencyUganda, Cambodia, Cameroon, Guinea, Vietnam, Zambia
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Adrian LacyUltragenyx Pharmaceutical IncCompletedGlucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)United States
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TakedaWithdrawnChronic Obstructive Pulmonary Disease (COPD) | Alpha1-Antitrypsin Deficiency
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Great Ormond Street Hospital for Children NHS Foundation...National Institute for Health Research, United Kingdom; The Freya FoundationRecruitingPyruvate Dehydrogenase Complex Deficiency | Pyruvate Dehydrogenase E1 Alpha Deficiency | Pyruvate Dehydrogenase E1-Beta Deficiency | Pyruvate Dehydrogenase E2 Deficiency | Pyruvate Dehydrogenase Phosphatase DeficiencyUnited Kingdom
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Ultragenyx Pharmaceutical IncTerminatedGlucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)United States, Spain, France, Germany, Italy, United Kingdom
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Gerard TurinoCompletedEmphysema | Alpha 1-Antitrypsin DeficiencyUnited States, Canada
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Medical University of South CarolinaCSL BehringCompletedSmoking | Emphysema | Alpha-1 Antitrypsin Deficiency | COPDUnited States
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IpsenCompletedGrowth Disorders | Insulin-Like Growth Factor-1 DeficiencyUnited States
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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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Grifols Therapeutics LLCCompletedAlpha 1-Antitrypsin DeficiencyDenmark, Sweden, United Kingdom
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National Heart, Lung, and Blood Institute (NHLBI)CompletedLung Diseases, Obstructive | Emphysema | Alpha 1-Antitrypsin DeficiencyUnited States
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Novartis PharmaceuticalsCompletedMyelodysplastic Syndromes | Fanconi Syndrome | Anemia, Aplastic | Beta-thalassemia | Anemia, Diamond-BlackfanUnited States
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Permian Health Lung InstituteThe Gambia Committee on Traditional Practices Affecting the Health of Women...Not yet recruitingCOPD | Lung Function Decreased | Alpha 1-AntitrypsinGambia
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University of NottinghamNottingham University Hospitals NHS TrustRecruitingChronic Obstructive Pulmonary Disease | Alpha 1-Antitrypsin DeficiencyUnited Kingdom
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Assistance Publique - Hôpitaux de ParisUnknownEmphysema | Alpha 1-Antitrypsin DeficiencyFrance
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Inhibrx, Inc.Not yet recruitingEmphysema | Alpha 1-Antitrypsin Deficiency
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Inhibrx Biosciences, IncRecruitingEmphysema | Alpha 1-Antitrypsin DeficiencyUnited States, Australia, United Kingdom, New Zealand
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Schön Klinik Berchtesgadener LandUnknownChronic Obstructive Pulmonary Disease | Alpha 1-Antitrypsin DeficiencyGermany
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National Heart, Lung, and Blood Institute (NHLBI)CompletedLung Diseases | Chronic Obstructive Pulmonary Disease | Emphysema | Alpha-1 Antitrypsin Deficiency
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Alpha-1 FoundationCompletedChronic Obstructive Pulmonary Disease | Alpha-1 Antitrypsin DeficiencyUnited States, Puerto Rico
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Genzyme, a Sanofi CompanyCompletedCerebroside Lipidosis Syndrome | Glucosylceramide Beta-Glucosidase Deficiency Disease | Gaucher Disease Type I | Clucocerebrosidase Deficiency Disease | Gaucher Disease, Non-Neuronopathic FormUnited States
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University of FloridaTerminatedChronic Obstructive Pulmonary Disease | Alpha-1 Antitrypsin DeficiencyUnited 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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Instituto Nacional de Ciencias Medicas y Nutricion...SuspendedHIV-1-infection | Immune Deficiency | CD4+ T Lymphocytopenia | Immuno-senescenceMexico
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Genzyme, a Sanofi CompanyTerminatedFabry Disease | Alpha Galactosidase A DeficiencyUnited States, Austria, United Kingdom
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Vitaflo International, LtdWithdrawnEpilepsy | Glucose Transporter Type 1 Deficiency Syndrome | Ketogenic Dieting
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University of EdinburghNHS Lothian; DexCom, Inc.; Tandem Diabetes Care, Inc.; The Leona M. and Harry B...Active, not recruitingHypoglycemia | Type 1 Diabetes | Type 1 Diabetes Mellitus With Hypoglycemia | Hypoglycemia Unawareness | Glucagon Deficiency | Insulin HypoglycemiaUnited Kingdom
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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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Rambam Health Care CampusHadassah Medical Organization; Tel-Aviv Sourasky Medical Center; Soroka University...CompletedGVHD | Bone Marrow Transplant Failure | Alpha 1-AntitrypsinIsrael