Clinical Trials on Inborn Error of Bile Acid Metabolism
Total 3064 results
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University Hospital PadovaRecruitingHypertension | Essential Hypertension | Hyperaldosteronism | Aldosteronism | Conn Adenoma | Aldosterone Disorder | Aldosterone-producing Adenoma | Bilateral Adrenal HyperplasiaItaly
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Centre Hospitalier Universitaire de NīmesCompletedStroke | Hemorrhage | Cerebral Amyloid AngiopathyFrance
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Auburn UniversityCompletedAlzheimer Disease | Cerebral Amyloid AngiopathyUnited States
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NYU Langone HealthCompletedCystinuriaUnited States
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NYU Langone HealthNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)Completed
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Fairview University Medical CenterUnknownMucopolysaccharidosis I | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Metachromatic Leukodystrophy | Pulmonary Complications | Fucosidosis | Globoid Cell Leukodystrophy | Gaucher's Disease | Mannosidosis | I Cell DiseaseUnited States
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Advicenne PharmaNot yet recruitingCystinuriaFrance, Belgium
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Revive Therapeutics, Ltd.Unknown
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Imperial College LondonCompletedGestational Diabetes Mellitus | Intrahepatic Cholestasis of PregnancyUnited Kingdom
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Alnylam PharmaceuticalsActive, not recruitingTransthyretin Amyloidosis (ATTR) With CardiomyopathyUnited States, Australia, Brazil, Bulgaria, France, Italy, Japan, Mexico, Netherlands, Taiwan, United Kingdom, Belgium, Czechia, Denmark, New Zealand, Sweden, Korea, Republic of, Argentina, Chile, Hong Kong, Portugal
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Alnylam PharmaceuticalsApproved for marketingAmyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid Polyneuropathies
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Alnylam PharmaceuticalsActive, not recruitingTransthyretin Amyloidosis (ATTR) With CardiomyopathyUnited States, Poland, Argentina, Australia, Belgium, France, Germany, Japan, Netherlands, Portugal, Spain, Sweden, United Kingdom, Canada, Czechia, Denmark, Hungary, Ireland, Norway, Korea, Republic of, Austria, Croatia, Israel, Latvia, Lithuan... and more
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Altoosi University CollegeBabylon UniversityUnknownCerebral Palsy | Cancer | HIV/AIDS | Muscular Dystrophy | Cystic Fibrosis | Cardiac Anomaly | Batten's DiseaseIraq
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Intellia TherapeuticsRegeneron PharmaceuticalsRecruitingTransthyretin Amyloidosis (ATTR) With CardiomyopathyUnited States, Australia, United Kingdom, New Zealand, Canada
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Alnylam PharmaceuticalsCompletedAmyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid PolyneuropathiesUnited States, Canada, Argentina, Australia, Bulgaria, Cyprus, Italy, Japan, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Germany, France, Brazil, Turkey, Korea, Republic of, Taiwan, United Kingdom
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Cystinose StiftungLabor Becker & Kollegen, Munich, Germany; Screening Labor Hannover; Genetikum... and other collaboratorsCompletedSpinal Muscular Atrophy | Cystinosis | Cystinosis, NephropathicGermany
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Devicare S.L.Clever Instruments S.L.; Fundacio PuigvertUnknown
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Arbor Research Collaborative for HealthNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); University...SuspendedEnd Stage Liver Disease | Mitochondrial Diseases | Acute Liver Failure | Respiratory Chain Deficiencies, Mitochondrial | Disorder of Fatty Acid OxidationUnited States, Canada
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McGill University Health Centre/Research Institute...Canadian Institutes of Health Research (CIHR)RecruitingZellweger Spectrum DisorderCanada
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Masonic Cancer Center, University of MinnesotaRecruiting
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Assistance Publique - Hôpitaux de ParisBraintaleNot yet recruitingX-linked Adrenoleukodystrophy
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Minoryx Therapeutics, S.L.RecruitingCerebral Adrenoleukodystrophy (cALD)United States, Brazil, Argentina
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Poxel SANot yet recruiting
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bluebird bioCompletedCerebral Adrenoleukodystrophy (CALD)United States, France, Germany, Italy, Netherlands, United Kingdom
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Stanford UniversityNational Institute of Neurological Disorders and Stroke (NINDS); Hugo W. Moser... and other collaboratorsCompletedX-linked AdrenoleukodystrophyUnited States
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bluebird bioCompletedCerebral Adrenoleukodystrophy (CALD)United States, Germany, Argentina, Australia, France, United Kingdom
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Shenzhen Geno-Immune Medical InstituteUnknown
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NeuroVia, Inc.WithdrawnX-Linked AdrenoleukodystrophyAustralia, Argentina, United Kingdom, Colombia, Chile, France, Russian Federation, Ukraine
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Masonic Cancer Center, University of MinnesotaWithdrawnCerebral Adrenoleukodystrophy
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FDA Office of Orphan Products DevelopmentHugo W. Moser Research Institute at Kennedy Krieger, Inc.Completed
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Far Eastern Memorial HospitalNational Taiwan University Hospital; Fu Jen Catholic UniversityUnknownAdrenoleukodystrophyTaiwan
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Beijing Tiantan HospitalRecruiting
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Capital Medical UniversityWangjing Hospital, China Academy of Chinese Medical Sciences; Bejing Fengtai...RecruitingCerebral Amyloid Angiopathy | Intracerebral Hemorrhage LobarChina
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Minoryx Therapeutics, S.L.Active, not recruitingAdrenoleukodystrophySpain, United States, Hungary, France, Germany, Italy, Netherlands, United Kingdom
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National Taiwan University HospitalCompleted
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Masonic Cancer Center, University of MinnesotaWithdrawn
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University of MinnesotaNo longer availableX-linked AdrenoleukodystrophyUnited States
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MedDay Pharmaceuticals SACompletedAdrenoleukodystrophy | Adrenomyeloneuropathy | AMNFrance, Germany, Spain
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Sunnybrook Health Sciences CentreWithdrawnCerebral Amyloid AngiopathyCanada
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Eunice Kennedy Shriver National Institute of Child...Food and Drug Administration (FDA)TerminatedAdrenoleukodystrophy | AdrenomyeloneuropathyUnited States
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Academisch Medisch Centrum - Universiteit van Amsterdam...The Stop ALD FoundationCompletedAdrenomyeloneuropathy | X-linked AdrenoleukodystrophyNetherlands
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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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bluebird bioCenter for International Blood and Marrow Transplant ResearchRecruitingCerebral Adrenoleukodystrophy (CALD)United States
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University Hospital Inselspital, BerneRecruitingCerebral Small Vessel Diseases | Intracerebral Hemorrhage | CAA - Cerebral Amyloid AngiopathySwitzerland
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Poxel SANot yet recruiting
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Masonic Cancer Center, University of MinnesotaRecruitingCerebral Adrenoleukodystrophy | Adrenoleukodystrophy | ALD (Adrenoleukodystrophy)United States
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Onofre, Aurora Pujol, M.D.Completed
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Hugo W. Moser Research Institute at Kennedy Krieger...TerminatedAdrenoleukodystrophyUnited States
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Onofre, Aurora Pujol, M.D.Instituto de Salud Carlos III; Fundacion Hesperia; ELA España AssociationCompletedAdrenomyeloneuropathy | X-linked AdrenoleukodystrophySpain
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Onofre, Aurora Pujol, M.D.Ministerio de Sanidad, Servicios Sociales e Igualdad; Fundacion HesperiaCompleted