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Clinical Trials on Brain Diseases, Metabolic
Total 380587 results
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Columbia UniversityEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationMitochondrial Disease | Mitochondrial Disorders | Melas | Kearns Sayer | NARP | MNGIE | LHON | Mitochondrial Depletion Syndrome | Leigh's DiseaseUnited States
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Massachusetts General HospitalRecruiting
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CENTOGENE GmbH RostockWithdrawnSkeletal Abnormalities | Psychomotor RetardationGermany, India, Sri Lanka, Egypt
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Baylor College of MedicineSeattle Children's Hospital; University of California, San FranciscoCompleted
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Emma Marie Caroline SlackUniversity Children's HospitalCompletedInborn Errors of Metabolism | Propionic Aciduria | Methylmalonic Aciduria | Urea Cycle DisorderSwitzerland
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Forge Biologics, IncNot yet recruitingKrabbe DiseaseUnited States
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University of PittsburghNational Institute of Neurological Disorders and Stroke (NINDS)Active, not recruiting
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Forge Biologics, IncActive, not recruiting
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Kaleido BiosciencesTerminatedUrea Cycle DisorderBelgium, United States, Spain, United Kingdom, Germany, Switzerland, Turkey
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HLB Cell Co., Ltd.WithdrawnUrea Cycle DisorderKorea, Republic of
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Horizon Therapeutics, LLCCompletedUrea Cycle DisorderUnited States, Canada
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Rabin Medical CenterGenzyme, a Sanofi CompanyUnknown
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingGaucher Disease Type 1United States
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Children's National Research InstituteActelionCompletedSandhoff Disease | GM2 Gangliosidoses | Tay-SachsUnited States
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University of MinnesotaRecruitingInflammation | Oxidative Stress | Gaucher Disease Type IUnited States
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National Institute of Neurological Disorders and...Completed
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GeneCradle IncRecruitingPompe Disease (Late-onset)China
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GeneCradle IncRecruitingPompe Disease Infantile-OnsetChina
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Seventh Medical Center of PLA General HospitalGeneCradle Therapeutics, IncRecruiting
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Duke UniversityCompleted
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Fundació Institut de Recerca de l'Hospital de la...Genzyme, a Sanofi CompanyUnknownPompe Disease | Late Onset Pompe DiseaseSpain
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Travere Therapeutics, Inc.Recruiting
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Hospital de Clinicas de Porto AlegreFundação de Amparo à Pesquisa do Estado do Rio Grande do Sul, BrazilSuspendedCBS DeficiencyBrazil
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Khondrion BVDrug Research Unit Ghent, BelgiumCompletedMitochondrial Disease | MELAS | Leigh Syndrome | LHON | Mitochondrial DNA tRNALeu(UUR) m.3243A<G MutationBelgium
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Swedish Orphan BiovitrumRecruitingHereditary Tyrosinemia, Type IChina
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedTay Sachs Disease | Sandhoff Disease | Late Onset Tay Sachs DiseaseUnited States
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Istanbul University - Cerrahpasa (IUC)CompletedUrea Cycle Disorder | Lysinuric Protein IntoleranceTurkey
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BioMarin PharmaceuticalCompletedHyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) DeficiencySweden, France, Italy, Spain, Germany, Netherlands, Austria, Slovakia, Portugal
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Andrea GropmanNational Center for Research Resources (NCRR); Rare Diseases Clinical Research... and other collaboratorsCompletedBrain Diseases, Metabolic, Inborn | Ornithine Transcarbamylase Deficiency | Urea Cycle DisorderUnited States
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SynlogicCompletedHealthy Volunteer | Urea Cycle DisorderUnited States
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Baylor College of MedicineCompletedUrea Cycle Disorders | Argininosuccinic Aciduria | Argininosuccinate Lyase DeficiencyUnited States
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Centre Hospitalier Universitaire de BesanconUnknownBiomarkers | Enteral Nutrition | Intensive Care Unit
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Ludwig-Maximilians - University of MunichCompletedPhenylketonuria | Nutritional DeficienciesGermany
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National Center for Research Resources (NCRR)Baylor College of MedicineUnknownAmino Acid Metabolism, Inborn ErrorsUnited States
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Arcturus Therapeutics, Inc.RecruitingOTC Deficiency | Ornithine Transcarbamylase Deficiency | OTCDSpain, Belgium, United Kingdom, Sweden
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Ultragenyx Pharmaceutical IncTerminatedOrnithine Transcarbamylase DeficiencyUnited States
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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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Forge Biologics, IncRecruitingKrabbe DiseaseUnited States
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CENTOGENE GmbH RostockWithdrawnKrabbe DiseaseEgypt, Germany, India, Sri Lanka
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Baylor College of MedicineRare Diseases Clinical Research Network; Neogenis LaboratoriesCompletedUrea Cycle Disorders, Inborn | Urea Cycle Disorder | Argininosuccinic Aciduria | Argininosuccinate Lyase DeficiencyUnited States
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SanofiActive, not recruitingGaucher's Disease Type III | Gaucher's Disease Type ICanada, Argentina, France, Italy, Japan, Russian Federation, Spain, Sweden, Turkey, United Kingdom
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PfizerNo longer availableGaucher DiseaseUnited States, Israel
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Dr. Anupam SehgalTaysha Gene Therapies, Inc.; GlycoNetActive, not recruitingInfantile GM2 Gangliosidosis (Disorder)Canada
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Astellas Gene TherapiesRecruitingPompe Disease (Late-onset)United States, Spain, France, Taiwan, Italy
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Amicus TherapeuticsCompletedPompe Disease (Late-onset)United States, Australia, Korea, Republic of, Japan, Taiwan, Belgium, Slovenia, New Zealand, United Kingdom, France, Argentina, Canada, Spain, Bosnia and Herzegovina, Germany, Sweden, Hungary, Austria, Bulgaria, Denmark, Greece, I... and more
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Duke UniversityCompleted
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Amicus TherapeuticsTerminatedLate-onset Pompe DiseaseUnited States, Canada, Australia, Belgium
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BioMarin PharmaceuticalTerminatedLate-onset Pompe DiseaseUnited States, United Kingdom, Germany, France, Italy, Portugal, Belgium, Netherlands
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Huashan HospitalNot yet recruitingPompe Disease (Late-onset)China
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Rambam Health Care CampusRecruitingPompe Disease Infantile-OnsetIsrael