Search clinical trials for: Inborn errors of Metabolism
Total 435667 results
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Amicus TherapeuticsRecruitingPompe Disease (Late-onset)United States, Japan, Taiwan, Australia, Canada, Italy, Germany
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Cambridge University Hospitals NHS Foundation TrustRecruitingDiabetes | Diet Modification | LipodystrophyUnited Kingdom
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University Hospital, MontpellierRecruiting
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Assistance Publique - Hôpitaux de ParisRecruitingX-linked HypophosphatemiaFrance
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Istanbul UniversityBiruni UniversityNot yet recruitingLipid Storage DiseaseTurkey
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University Hospital, AngersRecruiting
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Tehran University of Medical SciencesMashhad University of Medical Sciences; Kashan University of Medical SciencesRecruitingEffects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) (EMTISTD)Supportive Care | GM2 GangliosidosisIran, Islamic Republic of
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Masonic Cancer Center, University of MinnesotaRecruitingCerebral Adrenoleukodystrophy | Adrenoleukodystrophy | ALD (Adrenoleukodystrophy)United States
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Protecting Brains Saving FuturesNot yet recruitingBrain Injuries | Intraventricular Hemorrhage | Congenital Heart Disease | Hypoxic-Ischemic Encephalopathy | Newborn Morbidity | Inborn Errors of Metabolism | Extracorporeal Membrane Oxygenation Complication | Respiratory Complication | Hemodynamic Instability | Nosocomial Infection | Neonatal Seizure | Brain... and other conditionsBrazil
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University of California, San DiegoRocket Pharmaceuticals Inc.Recruiting
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Federico II UniversityRecruitingCongenital Adrenal HyperplasiaItaly
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Massachusetts General HospitalNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)RecruitingX-linked Hypophosphatemia | Hypophosphatemic Rickets | Hypophosphatemic Rickets, X-Linked DominantUnited States
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PerspectumRecruitingLiver Diseases | Cirrhosis | Primary Sclerosing Cholangitis | Autoimmune Hepatitis | Hemochromatosis | Non-Alcoholic Fatty Liver Disease | Non-Alcoholic Steatohepatitis | Primary Biliary Cirrhosis | Viral HepatitisNetherlands, United Kingdom
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Huilian ZhuNot yet recruiting
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Massachusetts General HospitalRecruiting
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Wuerzburg University HospitalCharite University, Berlin, Germany; University Hospital Muenster; Health Care...RecruitingQuality of Life | Adherence, Medication | Fabry Disease | Rare DiseasesGermany
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University of British ColumbiaCanadian Institutes of Health Research (CIHR); AllerGen NCE Inc.RecruitingExposure to Pollution | Glucocorticoid ResistanceCanada
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Ultragenyx Pharmaceutical IncPRA Health SciencesRecruitingSly Syndrome | Mucopolysaccharidosis VII | MPS VII | MPS 7United States, Germany, Brazil, Argentina, France, Netherlands, Portugal, Spain, Turkey
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TRPHARMKlinar CRO; Düzen Laboratories GroupRecruitingCerebrotendinous XanthomatosisTurkey
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Assistance Publique - Hôpitaux de ParisRecruiting
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University Of PerugiaRecruitingObesity | Ketosis | Lysosomal Acid Lipase Deficiency | Ketogenic DietingItaly
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University of UlmUniversity of Pisa; IRCCS Azienda Ospedaliero-Universitaria di Bologna; Medical... and other collaboratorsRecruitingLipodystrophy Acquired | Lipodystrophy CongenitalAustria, France, Germany, Hungary, Italy, Portugal, Spain, Netherlands, Russian Federation, Slovenia, Switzerland, Turkey, United Kingdom
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University of Texas Southwestern Medical CenterNational Institutes of Health Clinical Center (CC); University of Michigan; Children... and other collaboratorsNot yet recruitingCongenital Adrenal HyperplasiaUnited States
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Agios Pharmaceuticals, Inc.RecruitingPyruvate Kinase DeficiencyUnited States, Germany, Spain, Italy, United Kingdom, Czechia, France, Canada, Korea, Republic of, Netherlands, Turkey, Portugal, Denmark, Ireland, Switzerland, Thailand
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Washington University School of MedicineEunice Kennedy Shriver National Institute of Child Health and Human Development...RecruitingNiemann-Pick Disease, Type CUnited States
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GenethonRecruitingCrigler-Najjar SyndromeItaly, France, Netherlands
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Yassin Abdelghaffar Charity Center for Liver Disease...Society of Friends of Liver Patients in the Arab World (SLPAW)RecruitingHereditary Tyrosinemia, Type IEgypt
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The Hospital for Sick ChildrenChildren's Hospital of Philadelphia; Canadian Institutes of Health Research... and other collaboratorsRecruitingIdiopathic Infantile Hypercalcemia - Mild FormCanada
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Wuerzburg University HospitalAcademisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA); St George... and other collaboratorsRecruitingFabry Disease | Rare Diseases | Hypertrophic Cardiomyopathy | Fabry Disease, Cardiac VariantGermany
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Yale UniversityWilson Disease AssociationRecruiting
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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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Eunice Kennedy Shriver National Institute of Child...RecruitingBatten Disease | Juvenile Neuronal Ceroid Lipofuscinosis (CLN3)United States
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Children's Hospital of PhiladelphiaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)RecruitingGenetic Disease | Hypercalciuria | Idiopathic Infantile Hypercalcemia - Mild Form | Idiopathic Infantile Hypercalcaemia - Severe Form | Hypercalcemia, Idiopathic, of Infancy | Hypercalciuric HypercalcemiaUnited States
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Emily de los ReyesRecruitingBatten Disease | CLN6United States
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Cambridge University Hospitals NHS Foundation TrustMedical Research Council; National Institute for Health Research, United KingdomRecruitingGaucher Disease, Type III | Gaucher Disease, Type IUnited Kingdom
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CENTOGENE GmbH RostockAlnylam PharmaceuticalsRecruitingCardiomyopathies | Polyneuropathies | Transthyretin Amyloidosis | Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid PolyneuropathyGermany, Austria, Switzerland
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Yonsei UniversityRecruitingFabry DiseaseKorea, Republic of
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GOIZETBioMarin PharmaceuticalRecruiting
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Duke UniversityRecruiting
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University Hospital, LilleMinistry of Health, FranceRecruitingDiagnoses Disease | Peroxisomal DisordersFrance
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The University of Texas Health Science Center,...RecruitingLeigh Disease | Leigh Syndrome | Leigh's Necrotizing Encephalopathy | Subacute Necrotizing Encephalomyopathy | Subacute Necrotizing EncephalomyelopathyUnited States
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University of PittsburghNational Institute of Neurological Disorders and Stroke (NINDS); Rare Diseases...RecruitingPyruvate Dehydrogenase Complex Deficiency DiseaseUnited States
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Children's Hospital of PhiladelphiaEli Lilly and Company; University of Pennsylvania; Takeda; National Institute of... and other collaboratorsRecruitingMucopolysaccharidoses | Leukoencephalopathies | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2... and other conditionsUnited States
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Vanderbilt University Medical CenterHarvard UniversityRecruitingAlbright Hereditary Osteodystrophy | PseudohypoparathyroidismUnited States
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Travere Therapeutics, Inc.RecruitingNatural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)Homocystinuria Due to CBS DeficiencyIreland, Qatar, United States, United Kingdom
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State University of New York at BuffaloEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsRecruiting
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University of ThessalyNot yet recruiting
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Children's National Research InstituteBoston Children's HospitalRecruitingUrea Cycle DisordersUnited States
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Translational Research Center for Medical Innovation...RecruitingNeutral Lipid Storage DiseaseChina, United States, France, Austria, Germany, Italy, Japan, Netherlands, United Kingdom