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Search clinical trials for: Inborn Errors of Metabolism
Total 431502 results
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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 PharmaRecruitingGeneralized Arterial Calcification of Infancy | Autosomal Recessive Hypophosphatemic Rickets | Ectonucleotide Pyrophosphatase/phosphodiesterase1 DeficiencyUnited States, United Kingdom
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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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Immusoft of CA, Inc.RecruitingMucopolysaccharidosis IH/S | Mucopolysaccharidosis ISUnited States
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Novartis PharmaceuticalsRecruitingHeterozygous or Homozygous Familial HypercholesterolemiaNetherlands, Israel, Hungary, Italy, Germany, Spain, United States, France, Norway, Russian Federation, South Africa, Turkey, 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
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Olatec Therapeutics LLCRecruitingArthritis | Joint Pain | Gout | Gout Flare | Gouty Arthritis | Acute Gout Flare | Gout AttackUnited States, Spain, France
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Eunice Kennedy Shriver National Institute of Child...RecruitingNiemann-Pick Disease Type C1 | Juvenile Neuronal Ceroid Lipofuscinosis | Smith-Lemli-Opitz Syndrome | Creatine Transporter DeficiencyUnited States
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Fundación Española de Hematología y HemoterapíaRecruitingNiemann-Pick Diseases | Gaucher Disease | Splenomegaly | Acid SphingoMyelinase Deficiency | ASMDSpain
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Institute of Health Information and Statistics...Centre of Cardiovascular and Transplantation Surgery, Czech Republic; The Central... and other collaboratorsRecruitingFamilial HypercholesterolaemiaCzechia
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Masonic Cancer Center, University of MinnesotaRecruitingMucopolysaccharidosis Type I | Hematopoietic Cell TransplantationUnited States
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University of California, San FranciscoRecruitingMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI | Wolman Disease | Pompe Disease Infantile-Onset | Mucopolysaccharidosis IV A | Mucopolysaccharidosis VII | Neuronopathic Gaucher DiseaseUnited States
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Odense University HospitalNot yet recruitingFamilial HypercholesterolemiaDenmark
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Lille Catholic UniversityVariant Bio, Inc.RecruitingRenal Insufficiency, Chronic | GoutNew Caledonia
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Assistance Publique - Hôpitaux de ParisNot yet recruiting
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Ramsay Générale de SantéEuropean Clinical Trial Experts NetworkNot yet recruitingDialysis | Iron Deposition | Iatrogenic HemosiderosisFrance
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Sunshine Guojian Pharmaceutical (Shanghai) Co.,...Recruiting
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Eunice Kennedy Shriver National Institute of Child...RecruitingNiemann-Pick Disease, Type CUnited States
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LG ChemRecruitingHyperuricemia | Gout | Gout Flare | TophiUnited States
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University of MinnesotaRecruitingGaucher Disease, Type 3United States
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LG ChemRecruitingHyperuricemia | Gout | Gout Flare | TophiUnited States
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BioMarin PharmaceuticalSyneos HealthRecruitingPhenylketonuria, MaternalGermany, United States, Italy
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Massachusetts General HospitalPfizer; Akcea Therapeutics; Alnylam Pharmaceuticals; Eidos Therapeutics, a BridgeBio...RecruitingHeart Failure | Amyloidosis | Heart Failure, Diastolic | Transthyretin AmyloidosisUnited States
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University of ThessalyNot yet recruitingOxidative Stress | Carbohydrate Metabolism Disorder | G6PD Deficiency
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Marcello ArcaPfizer; Göteborg University; University of HelsinkiNot yet recruitingFamilial Hypobetalipoproteinemia | Familial Hypobetalipoproteinemia - Heterozygous Form