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Search clinical trials for: inborn errors of metabolism
Total 428106 results
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AstraZenecaRecruiting
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Fondazione SISA (Societa Italiana per lo Studio...RecruitingFamilial Hypercholesterolemia | Genetic DisorderItaly
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Cliniques universitaires Saint-Luc- Université...RecruitingRare Diseases | Inborn Errors of MetabolismBelgium
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Alnylam PharmaceuticalsNot yet recruitingHereditary Amyloidosis, Transthyretin-Related | Asymptomatic Carrier StateFrance
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John Paul II Hospital, KrakowRecruitingTransthyretin AmyloidosisPoland
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Fondation Ophtalmologique Adolphe de RothschildNot yet recruitingAlbinism, Ocular
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AstraZenecaRecruitingTransthyretin Amyloidosis Cardiomyopathy, Heart Failure With Preserved Ejection FractionRussian Federation
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Jirair Krikor BedoyanUltragenyx Pharmaceutical IncRecruitingPyruvate Dehydrogenase Complex DeficiencyUnited States
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Rigshospitalet, DenmarkNot yet recruitingBrain Diseases | Genetic Diseases, Inborn | Metabolism, Inborn Errors | Brain Diseases, Metabolic | Phenylketonurias | Brain Diseases, Metabolic, Inborn | Amino Acid Metabolism, Inborn Errors | Metabolic DiseaseDenmark
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Medical University of ViennaUniversity of Vienna; Griffith UniversityRecruiting
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NGGT (Suzhou) Biotechnology Co., Ltd.Not yet recruitingPhenylketonurias
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Lundquist Institute for Biomedical Innovation at...Not yet recruitingMucopolysaccharidosis III | Sanfilippo SyndromeUnited States
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Maastricht University Medical CenterRecruitingQuality of Life | Tuberous Sclerosis | Basal Cell Nevus Syndrome | Cutis Laxa | Epidermolysis Bullosa | Ichthyosis | Ectodermal Dysplasia | Albinism | Birt-Hogg-Dube Syndrome | Palmoplantar KeratosesNetherlands
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Hospital do CoracaoUniversity of Sao Paulo; Instituto Dante Pazzanese de Cardiologia; National Institute...Not yet recruiting
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Chiesi Farmaceutici S.p.A.ICON plcNot yet recruiting
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Caroline Michaela KistorpSanofiNot yet recruitingChronic Kidney Diseases | Fabry DiseaseDenmark
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National Taiwan University HospitalRecruiting
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Forge Biologics, IncNot yet recruiting
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BioMarin PharmaceuticalRecruitingPhenylketonuria (PKU)United States
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PTC TherapeuticsNot yet recruitingPhenylketonuriaUnited States
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Shanghai Institute Of Biological ProductsThe First Affiliated Hospital of Henan University of Science and Technology; First Affiliated Hospital Bengbu Medical College and other collaboratorsRecruiting
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metaX Institut fuer Diatetik GmbHGreat Ormond Street Hospital for Children NHS Foundation Trust; Birmingham...Not yet recruitingAlkaptonuria | Homocystine; Metabolic Disorder | Tyrosinemia, Type I | Tyrosinemia, Type II | Tyrosinemia, Type III | MSUD (Maple Syrup Urine Disease)
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Suzhou Municipal HospitalNot yet recruitingFamilial Hypercholesterolemia | Refractory Hypercholesterolemia
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Steen Hvitfeldt PoulsenNovo Nordisk A/SRecruitingQuality of Life | Transthyretin Amyloidosis | Transthyretin Amyloid Cardiomyopathy | Wild-Type Transthyretin-Related (ATTR)AmyloidosisDenmark
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Ondokuz Mayıs UniversityRecruitingQuality of Life | Urinary Incontinence | Menopause | Wolman Disease | Psychological Well-BeingTurkey
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Assistance Publique - Hôpitaux de ParisNot yet recruiting
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Novilla PharmaceuticalsNot yet recruitingAcute Pain | Gout Flare | Acute Gouty ArthritisAustralia
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Novartis PharmaceuticalsRecruitingHypercholesterolaemia | Familial HypercholesterolaemiaJapan
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Xinhua Hospital, Shanghai Jiao Tong University...Shanghai Vitalgen BioPharma Co., Ltd.RecruitingType II Gaucher DiseaseChina
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UniQure Biopharma B.V.Not yet recruiting
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Sunshine Guojian Pharmaceutical (Shanghai) Co.,...Recruiting
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S.LAB (SOLOWAYS)Center of New Medical TechnologiesRecruiting
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Leadiant Biosciences, Inc.Not yet recruitingCerebrotendinous Xanthomatoses
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Brunel UniversityRoyal Free Hospital NHS Foundation TrustRecruiting
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Chung-Hsing WangSanofiNot yet recruiting
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iECURE, Inc.RecruitingUrea Cycle Disorders, Inborn | Ornithine Transcarbamylase Deficiency | Ornithine Transcarbamylase Deficiency Disease | Ornithine Carbamoyltransferase Deficiency (Disorder)United Kingdom
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Hospices Civils de LyonNot yet recruitingX-Linked Hypophosphatemic Rickets
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Travere Therapeutics, Inc.Recruiting
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UNICANCERServierNot yet recruitingMetastatic Colorectal Cancer | Metastatic Gastroesophageal Adenocarcinoma | DPD Deficiency
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Horizon Therapeutics Ireland DACRecruiting
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Swedish Orphan BiovitrumRecruitingHereditary Tyrosinemia, Type IChina
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Hospices Civils de LyonRecruitingPatients With PH1 Treated With Lumasiran in FranceFrance
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University of CambridgeSanofiRecruiting
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bluebird bioCenter for International Blood and Marrow Transplant ResearchRecruitingCerebral Adrenoleukodystrophy (CALD)United States
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Shanghai Vitalgen BioPharma Co., Ltd.Not yet recruitingGlutaric Acidemia Type I | Glutaric Aciduria Type IChina
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University of PennsylvaniaRecruitingLCAT DeficiencyUnited States
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Rocket Pharmaceuticals Inc.Recruiting
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Hospital Universitari Vall d'Hebron Research InstituteErasme University Hospital; Cyprus Institute of Neurology and Genetics; EuroBloodNet...RecruitingIron Metabolism Disorders | Sickle Cell Disease | Thalassemia | Hemolytic; Anemia, Hereditary, Due to Enzyme Disorder | Anemia Due to Membrane Defect | CDA | Sideroblastic Anemia | Constitutional Aplastic Anemia | Hereditary AnemiaSpain
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Diakonhjemmet HospitalRecruiting
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Children's Hospital of Fudan UniversityNot yet recruiting