Clinical Trials on Urea Cycle Disorders, Inborn
Total 7070 results
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Beijing Children's HospitalPeking University First Hospital; Xijing Hospital; Xiangya Hospital of Central... and other collaboratorsRecruitingEpilepsy | Tuberous Sclerosis ComplexChina
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Paul SzabolcsRecruitingSickle Cell Anemia | Diamond-blackfan Anemia | Beta-thalassemia MajorUnited States
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University Hospital TuebingenRecruitingHereditary Spastic Paraplegia | Hereditary, Spastic Paraplegia, Autosomal DominantGermany
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Otsuka Canada Pharmaceutical Inc.RecruitingAutosomal Dominant Polycystic Kidney Disease (ADPKD)Canada
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Spectrum Health HospitalsVan Andel Research InstituteRecruitingNeurofibromatosis Type 1 | Plexiform Neurofibromas | Cutaneous NeurofibromasUnited States
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Institut Cancerologie de l'OuestRecruiting
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Teachers College, Columbia UniversityActive, not recruitingSpinocerebellar Ataxia Type 3 | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6 | Spinocerebellar Ataxia Type 7United States
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Scholar Rock, Inc.CompletedNeuromuscular Diseases | Neuromuscular Manifestations | Atrophy | Muscular Atrophy | Spinal Muscular Atrophy | Muscular Atrophy, Spinal | Spinal Muscular Atrophy Type 3 | SMA | Spinal Muscular Atrophy Type 2United States, Spain, Italy, Netherlands
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University of NottinghamNottingham University Hospitals NHS Trust; Action for A-TCompleted
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AstraZenecaMerck Sharp & Dohme LLCActive, not recruitingNeurofibromatosis Type 1United States, Spain, Poland, Russian Federation
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University Hospital, Basel, SwitzerlandEnrolling by invitationSpinal Muscular Atrophy Type II | Spinal Muscular Atrophy Type IIISwitzerland
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University of Alabama at BirminghamCompletedNeurofibromatosis | Plexiform Neurofibromas | NF1United States
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Aucta Pharmaceuticals, IncTerminatedTuberous Sclerosis | Angiofibroma of FaceUnited States, China
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University of British ColumbiaMHE CoalitionCompletedExostoses, Multiple HereditaryCanada
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Enzyvant Therapeutics GmBHNational Institute of Allergy and Infectious Diseases (NIAID); Eunice Kennedy... and other collaboratorsCompletedDiGeorge Syndrome | Hypoparathyroidism | Complete DiGeorge SyndromeUnited States
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Assistance Publique - Hôpitaux de ParisCompleted
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University of NottinghamNottingham University Hospitals NHS Trust; Royal Papworth HospitalActive, not recruitingAtaxia TelangiectasiaUnited Kingdom
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Hospices Civils de LyonWithdrawnFacial AngiofibromasFrance
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National Cancer Institute (NCI)CompletedPlexiform Neurofibromas | NF1United States
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University of Alabama at BirminghamThe University of Texas Health Science Center, Houston; University of California... and other collaboratorsCompletedTuberous Sclerosis ComplexUnited States
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Shriners Hospitals for ChildrenUniversity of UtahCompletedNeurofibromatosis Type 1 | Tibial DysplasiaUnited States
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Lei LiUnknownColorectal Neoplasms, Hereditary Nonpolyposis | Endometrial NeoplasmsChina
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Novartis PharmaceuticalsCompletedTuberous Sclerosis Complex-associated Refractory SeizuresUnited States, Colombia, Belgium, France, Japan, Taiwan, Turkey, Spain, Thailand, Korea, Republic of, Australia, Hungary, United Kingdom, Canada, Norway, Germany, Italy, Russian Federation, Denmark, Argentina, Netherlands, Greece, P... and more
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Flex Pharma, Inc.TerminatedCharcot-Marie-Tooth DiseaseUnited States
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Indiana UniversityPfizer; United States Department of DefenseTerminatedNeurofibromatosis | Plexiform Neurofibromas | NF1United States
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Johns Hopkins UniversityCompleted
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University of Alabama at BirminghamNational Cancer Institute (NCI); Children's Hospital of Philadelphia; Washington... and other collaboratorsCompletedNeurofibromatosis Type 1United States
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National Institute of Allergy and Infectious Diseases...TerminatedGrowth Failure | X-linked Severe Combined Immunodeficiency (XSCID) | Growth Hormone ResistenceUnited States
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Ramathibodi HospitalAstraZenecaCompletedSpinobulbar Muscular Atrophy | Kennedy's DiseaseThailand
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Johann Wolfgang Goethe University HospitalCompletedAtaxia Telangiectasia
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Radboud University Medical CenterCompletedAnterior Cutaneous Nerve Entrapment Syndrome (ACNES)Netherlands
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Wayne State UniversityMuscular Dystrophy Association; Charcot-Marie-Tooth AssociationCompleted
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University of GenovaUnknownCharcot-Marie-Tooth Disease | Charcot-Marie-Tooth Disease Type 1AItaly
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National Institute of Neurological Disorders and...CompletedSpinal and Bulbar Muscular Atrophy | Kennedy's DiseaseUnited States
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WestatNational Institute of Neurological Disorders and Stroke (NINDS)TerminatedSpinal Muscular Atrophy Type II | Spinal Muscular Atrophy Type IIIUnited States
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Sheba Medical CenterUnknownAtaxia TelangiectasiaIsrael
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Sheba Medical CenterUnknownAtaxia TelangiectasiaIsrael
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National Heart, Lung, and Blood Institute (NHLBI)RecruitingPneumothorax | Tuberous Sclerosis | Lung Disease | LymphangioleiomyomatosisUnited States
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Elpida Therapeutics SPCNot yet recruitingNeuromuscular Diseases | Peripheral Neuropathy | Neuro-Degenerative Disease
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Shanghai Fosun Pharmaceutical Industrial Development...RecruitingNeurofibromatosis 1 | NF1 | Plexiform NeurofibromaChina
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Istanbul Medipol University HospitalIstanbul UniversityRecruitingNeuromuscular Diseases | Spinal Muscular Atrophy Type 3Turkey
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Katarzyna KotulskaRecruiting
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Darcy KruegerRecruitingEpilepsy | Tuberous Sclerosis ComplexUnited States
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Centre Hospitalier Intercommunal CreteilEtablissement Français du Sang; Centre National de la Recherche Scientifique...RecruitingSickle Cell Disease | Stenosis | Cerebral IschemiaFrance
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University Hospital, AngersRecruitingCharcot-Marie-Tooth Disease Type 2AFrance
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Hospital General Universitario Gregorio MarañonHospital Clinic of Barcelona; University of SalamancaRecruitingStroke | Dilated Cardiomyopathy | Hypertrophic Cardiomyopathy | Thrombosis CardiacSpain
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Hôpital le VinatierRecruiting
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Marinus PharmaceuticalsEnrolling by invitationTuberous Sclerosis ComplexUnited States, Spain, United Kingdom, China, Italy, France, Israel, Germany, Canada, Australia
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AstraZenecaMerck Sharp & Dohme LLCActive, not recruitingNeurofibromatosis 1 | Plexiform Neurofibroma (PN)United States, Canada, France, Italy, Germany, China, United Kingdom, Spain, Japan, Russian Federation, Australia, Poland, Brazil
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Oregon Health and Science UniversityActive, not recruitingAnterior Cutaneous Nerve Entrapment SyndromeUnited States