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Search clinical trials for: Inborn errors of metabolism
Total 433186 results
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Ionis Pharmaceuticals, Inc.RecruitingAlexander DiseaseUnited States, United Kingdom, Australia, Canada, Israel, Italy, Japan, Netherlands
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Hospital Clinico Universitario de SantiagoAlvaro Hermida (Co-IP); Susana Belen Bravo; Maria Teresa Cardoso; Cristobal Colon... and other collaboratorsRecruiting
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SanofiRecruitingGlycogen Storage Disease Type IIBelgium, United States, Italy, Spain, Taiwan, United Kingdom, Netherlands, France, Germany
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SanofiRecruitingAcid SphingoMyelinase Deficiency | Gaucher Disease, SplenomegalyFrance
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Assistance Publique - Hôpitaux de ParisRecruitingChronic Kidney Disease | Gout | Renal TransplantationFrance
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Monash University MalaysiaRecruiting
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Hospital Ruber InternacionalRecruitingRefractory Epilepsy | SLC35A2-CDG - Solute Carrier Family 35 Member A2 Congenital Disorder of GlycosylationSpain
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Myrtelle Inc.RecruitingCanavan DiseaseUnited States
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Albina Nowak, MDSwiss National Science FoundationRecruiting
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University of PennsylvaniaUniversity of Amsterdam; University of Cape Town; University of Witwatersrand...RecruitingHomozygous Familial HypercholesterolemiaUnited States, Netherlands, South Africa
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Amicus TherapeuticsRecruitingGlycogen Storage Disease Type II Infantile OnsetUnited States, Taiwan, France, Germany, Italy, United Kingdom
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Assiut UniversityNot yet recruitingPsychiatric Problem | Wolman Disease
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Adrenas Therapeutics IncRecruitingCongenital Adrenal HyperplasiaUnited States
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Rennes University HospitalRecruiting
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Shrouk Shaban BakrRecruiting
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Sixth Affiliated Hospital, Sun Yat-sen UniversityNot yet recruitingInflammatory Bowel Diseases | Crohn Disease | Polyunsaturated Acid Lipidosis
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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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M.A.G.I.C. Clinic LTDHanalytics Solutions Inc.RecruitingMitochondrial Diseases | Fabry Disease | Metabolic Disease | Gaucher Disease | Pompe DiseaseCanada
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Rambam Health Care CampusRecruitingPompe Disease Infantile-OnsetIsrael
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Assistance Publique - Hôpitaux de ParisRecruitingHyperuricemia | Gout | Inflammatory Rheumatism | Posology AdjustmentFrance
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Institut PasteurInstitut Pasteur de la Guyane; Centre Hospitalier Andrée Rosemon de CayenneRecruiting
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Leipzig University Medical CenterRecruitingX-linked AdrenoleukodystrophyGermany
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National Medical Research Center for Therapy and...Moscow State University of Medicine and DentistryRecruitingMedication Adherence | Adherence, Medication | Treatment Adherence | Familial Hypercholesterolemia | Motivational Interviewing | Adherence, Patient | Treatment Adherence and Compliance | Patient Compliance | Adherence | Hypercholesterolemia, Familial | Patient Adherence | Hypercholesterolemia, Autosomal Dominant and other conditionsRussian Federation
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Shenzhen Second People's HospitalNot yet recruitingCerebral Amyloid Angiopathy
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University Hospital, LilleRecruiting
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Yonsei UniversityRecruitingFabry DiseaseKorea, Republic of
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Duke UniversitySanofiRecruiting
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Polaryx Therapeutics, Inc.Not yet recruitingJuvenile Neuronal Ceroid Lipofuscinosis
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SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt)RecruitingGangliosidoses | Galactosialidosis | Sialidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Morquio B Disease | Gm2-Gangliosidosis, Variant B1 | GM2 Activator DeficiencyGermany
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Baylor College of MedicineChildren's Hospital of Philadelphia; Children's Hospital Colorado; Seattle Children... and other collaboratorsRecruitingOrnithine Transcarbamylase Deficiency | Urea Cycle Disorder | Argininosuccinic Aciduria | Hyperargininemia | Citrullinemia 1 | ARGI Deficiency | ASL Deficiency | ASS DeficiencyUnited States
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Universitätsklinikum Hamburg-EppendorfRecruitingBatten Disease | Neuronal Ceroid Lipofuscinosis | CLN1 Disease | CLN2 Disease | CLN3 Disease | CLN4 Disease | CLN5 Disease | CLN6 Disease | CLN7 Disease | CLN8 Disease | CLN10 Disease | CLN11 Disease | CLN12 Disease | CLN13 Disease | CLN14 DiseaseGermany
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National Taiwan University HospitalRecruitingCerebral Amyloid Angiopathy | Intracerebral HemorrhageTaiwan
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Children's National Research InstituteNational Center for Advancing Translational Sciences (NCATS)RecruitingHypoxic-Ischemic Encephalopathy | Urea Cycle Disorder | Organic Acidemia | Fatty Acid Oxidation Disorder | Maple Syrup Urine Disease | Glutaric Acidemia IUnited States
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Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyRecruitingEnd Stage Renal Disease | Primary Hyperoxaluria Type 2 | Primary Hyperoxaluria Type 1Germany, Spain, United States, United Kingdom, Italy, United Arab Emirates, Lebanon, Morocco, France, Romania
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JCR Pharmaceuticals Co., Ltd.RecruitingMucopolysaccharidosis IIUnited States, Germany, Spain, France, Brazil, Argentina, Turkey, United Kingdom, Poland, Italy
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Nemours Children's ClinicRhizoKids InternationalRecruitingRCDP - Rhizomelic Chondrodysplasia Punctata | RCDP1 | RCDP2 | RCDP3 | RCDP4 | RCDP5United States
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Vanderbilt University Medical CenterRecruitingPseudohypoparathyroidism Type 1a | Albright Hereditary Osteodystrophy | PseudohypoparathyroidismUnited States
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University of ChicagoRecruitingBreast Cancer | Head and Neck Cancer | Gastrointestinal Cancer | Dihydropyrimidine Dehydrogenase Deficiency | UGT1A1 Gene MutationUnited States
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Vivet Therapeutics SASRecruitingWilson's DiseaseUnited States, Denmark, Germany, United Kingdom
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Shanghai Jiao Tong University School of MedicineNot yet recruitingCongenital Adrenal HyperplasiaChina
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University of California, San FranciscoDuke UniversityRecruitingWolman Disease | MPS IVA | Pompe Disease Infantile-Onset | Gaucher Disease, Type 2 | MPS VI | MPS I | Gaucher Disease, Type 3 | MPS II | Mps VIIUnited States
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Children's Hospital of Fudan UniversityNot yet recruitingFamilial Hypercholesterolemia
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Liverpool University Hospitals NHS Foundation TrustRecruitingAlkaptonuria | Homogentisic Acid | Homogentisate Dioxygenase | Alkaptonuria Severity Score IndexUnited Kingdom
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University Hospital, BordeauxRecruiting
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Serhat Gumrukcu, MD PhDFrida Therapeutics LLCRecruiting
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dr. Laura C. G. de Graaff-HerderRecruitingDisorders of Sex Development | Congenital Adrenal Hyperplasia | Tuberous Sclerosis | Kallmann Syndrome | Prader-Willi Syndrome | Neurofibromatosis | Rett Syndrome | 22q11 Deletion Syndrome | Turner Syndrome | Noonan Syndrome | Allan-Herndon-Dudley Syndrome | Saethre-Chotzen Syndrome | Congenital Hypopituitarism | Cornelia... and other conditionsNetherlands
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Shanghai Yueyang Integrated Medicine HospitalNot yet recruitingGouty Arthritis
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Boston Children's HospitalWellth Inc.; MMAS-8 with permission from Dr Donald Morisky. as written belowRecruitingAdherence, Medication | Familial HypercholesterolemiaUnited States
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Duke UniversityKriya TherapeuticsRecruitingGlycogen Storage Disease VI | GLYCOGEN STORAGE DISEASE IXa1 | GLYCOGEN STORAGE DISEASE IXa2 | Glycogen Storage Disease IXB | Glycogen Storage Disease IXC | GSD 9 (All Subtypes) | GSD 6United States
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Institut National de la Santé Et de la Recherche...Not yet recruitingFabry Disease