Search clinical trials for: Inborn errors
Total 15924 results
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University of LausanneRecruiting
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Minoryx Therapeutics, S.L.RecruitingCerebral Adrenoleukodystrophy (cALD)United States, Brazil, Argentina
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Assiut UniversityNot yet recruiting
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Jiangsu Atom Bioscience and Pharmaceutical Co.,...RecruitingGoutUnited States, Georgia, Australia, Guatemala, Taiwan
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JW PharmaceuticalRecruitingGoutKorea, Republic of
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Katarzyna HolcmanRecruitingTransthyretin AmyloidosisPoland
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BioMarin PharmaceuticalRecruitingPhenylketonuria (PKU)United States, Germany, Italy
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Qianfoshan HospitalRecruitingIron Deficiency Anemia of Pregnancy | Iron Storage DiseaseChina
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RenJi HospitalWestlake TherapeuticsNot yet recruiting
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Central Hospital, Nancy, FranceNot yet recruitingAlzheimer Disease | Homocystinemia
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Assiut UniversityNot yet recruitingClinical Outcome in WILSON Disease
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GeneCradle IncRecruitingPompe Disease Infantile-OnsetChina
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Massachusetts General HospitalRecruiting
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Jnana TherapeuticsRecruitingPhenylketonuriaUnited States, Australia
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University Hospital Inselspital, BerneRecruitingCerebral Small Vessel Diseases | Intracerebral Hemorrhage | CAA - Cerebral Amyloid AngiopathySwitzerland
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ModernaTX, Inc.RecruitingPropionic AcidemiaUnited States, Spain, Netherlands, France, United Kingdom, Australia, Canada, Italy
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Fondazione Policlinico Universitario Agostino Gemelli...Recruiting
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Odense University HospitalRecruitingAtherosclerosis | Familial HypercholesterolemiaDenmark
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Centre Hospitalier Saint Joseph Saint Luc de LyonRecruitingGout ArthritisFrance
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Forge Biologics, IncRecruitingKrabbe DiseaseUnited States
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Inozyme PharmaRecruitingPseudoxanthoma Elasticum | Generalized Arterial Calcification of Infancy | Autosomal Recessive Hypophosphatemic Rickets | Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency | ATP-Binding Cassette Subfamily C Member 6 DeficiencyUnited States, United Kingdom, Spain
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University of AberdeenNot yet recruitingCancer | Sickle Cell Disease | Immunosuppression | Inborn Errors of Metabolism | Organ Transplant | COPD Asthma
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University Hospital Inselspital, BerneRecruitingCerebral Amyloid Angiopathy | Cadasil | Small Vessel Cerebrovascular Disease | CAA - Cerebral Amyloid AngiopathySwitzerland
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Amydis Inc.National Institutes of Health (NIH); National Institute on Aging (NIA)Recruiting
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Albina Nowak, MDNot yet recruiting
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Chiesi Farmaceutici S.p.A.ICON plcRecruiting
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Lysosomal and Rare Disorders Research and Treatment...Team SanfilippoRecruitingSanfilippo Syndrome | MPS3United States
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University Medicine GreifswaldWuerzburg University Hospital; University of GöttingenRecruiting
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Mackay Memorial HospitalRecruiting
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Intellia TherapeuticsRecruitingTransthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy | Wild-Type Transthyretin Cardiac AmyloidosisUnited Kingdom, New Zealand, Sweden
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Children's Hospital Medical Center, CincinnatiSanofiRecruitingFabry DiseaseUnited States
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University Hospital, BordeauxRecruitingIntellectual Disability | Cystic Fibrosis | Neurodegeneration With Brain Iron Accumulation (NBIA) | Congenital Heart Defect | Albinism | Rubinstein-Taybi Syndrome | Periventricular Nodular HeterotopiaFrance
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Shanton Pharma Co., Ltd.RecruitingGoutUnited States, Puerto Rico
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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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National Cancer Institute (NCI)RecruitingFanconi Anemia | Inherited Bone Marrow Failure SyndromeUnited States
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Immusoft of CA, Inc.RecruitingMucopolysaccharidosis IH/S | Mucopolysaccharidosis ISUnited States
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Novartis PharmaceuticalsRecruitingHeterozygous or Homozygous Familial HypercholesterolemiaNetherlands, Israel, Hungary, Italy, Germany, Turkey, Spain, United States, France, Norway, Russian Federation, South Africa, United Kingdom, Canada, Switzerland, Brazil, Lebanon, Slovenia, Poland, Taiwan, Malaysia
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Leiden University Medical CenterRecruiting
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Yonsei UniversityRecruitingFabry DiseaseKorea, Republic of
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University Children's Hospital, ZurichRecruitingUrea Cycle DisordersSwitzerland
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H. Lundbeck A/SRecruitingCongenital Adrenal HyperplasiaUnited Kingdom
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TakedaNot yet recruiting
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Purdue UniversityRecruitingLactose IntoleranceUnited States
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Purdue UniversityRecruiting
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University of ManchesterManchester University NHS Foundation Trust; Great Ormond Street Hospital for... and other collaboratorsRecruiting
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InventisBio Co., LtdRecruiting
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National Institutes of Health Clinical Center (CC)RecruitingCongenital Adrenal HyperplasiaUnited States
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Purdue UniversityRecruitingLactose IntoleranceUnited States
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Ionis Pharmaceuticals, Inc.RecruitingPelizaeus-Merzbacher DiseaseFrance, Australia, Germany, Netherlands