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Clinical Trials on Autosomal Recessive Disorder (Genetic Carriers of PKU) in United Kingdom
Total 580 results
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National Institute of Diabetes and Digestive and...RecruitingCystic Fibrosis | Turner Syndrome | Congenital Hepatic Fibrosis | Immunologic Deficiency Syndrome | Idiopathic Non-Cirrhotic Portal HypertensionUnited States
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New York Medical CollegeCompletedLymphoma | Leukemia | Sickle Cell Disease | Histiocytosis | Immunodeficiencies | Inborn Errors of Metabolism | Bone Marrow Failure | Beta ThalassemiaUnited States
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Joanne Kurtzberg, MDAldagenCompletedAnemia, Aplastic | MDS | Inborn Errors of Metabolism | Congenital Marrow Failure | Congenital Immunodeficiency SyndromeUnited States
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Eunice Kennedy Shriver National Institute of Child...RecruitingTurner Syndrome | Galactosemia | Ovarian Disfunction | Post-menarcheal Adolescents | Variations in Sex Characteristics | Differences in Sex DevelopmentUnited States
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University of MinnesotaCompletedDisorders of Sex Development | Urogenital Abnormalities | Congenital Adrenal Hyperplasia | Genetic Diseases, Inborn | Hyperplasia | Adrenocortical Hyperfunction | Congenital Disorders | Hydrocortisone | Adrenal Hyperplasia | Steroid Metabolic Diseases, Inborn | Adrenal Gland DiseaseUnited States
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Massachusetts General HospitalCompleted
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Children's Hospital Los AngelesCompletedGranuloma | Anemia | Thrombocytopenia | Neutropenia | Sickle Cell Disease | Thalassemia | Niemann-Pick Disease | Wiskott-Aldrich Syndrome | Osteopetrosis | Fucosidosis | Chediak Higashi Syndrome | Hurler DiseaseUnited States
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National Institutes of Health Clinical Center (CC)RecruitingCongenital Adrenal HyperplasiaUnited States
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Eunice Kennedy Shriver National Institute of Child...CompletedCongenital Adrenal Hyperplasia (CAH)United States
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University of Massachusetts, WorcesterEnrolling by invitationMotor Neuron Disease | Amyotrophic Lateral Sclerosis | Muscular Dystrophy | Frontotemporal Dementia | Miyoshi Myopathy | Amyotrophic Lateral Sclerosis, Sporadic | PLS | Lou Gehrigs Disease | Familial Disease | Amyotrophic Lateral Sclerosis With DementiaUnited States
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Johns Hopkins UniversityNational Cancer Institute (NCI); National Institutes of Health (NIH); ChiRhoClin...RecruitingLynch Syndrome | Pancreas Cancer | Gene Mutation | Peutz-Jeghers Syndrome (PJS) | Germline Mutation CarrierUnited States
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Spruce BiosciencesCompletedCongenital Adrenal Hyperplasia | CAH - Congenital Adrenal HyperplasiaUnited States
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Adrenas Therapeutics IncRecruitingCongenital Adrenal HyperplasiaUnited States
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University of California, Los AngelesUniversité de MontréalWithdrawnStroke | Epilepsy | Tuberous Sclerosis | Sturge-Weber Syndrome | Focal Cortical Dysplasia | Hemimegalencephaly | Polymicrogyria | Rasmussen Encephalitis | Gliosis | Tumor, BrainUnited States, Canada
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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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Neurocrine BiosciencesActive, not recruitingCongenital Adrenal HyperplasiaUnited States, Spain, Belgium, Austria, Bulgaria, Canada, Czechia, France, Germany, Greece, Israel, Italy, Netherlands, Poland, Portugal, Serbia, Sweden, United Kingdom
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SWOG Cancer Research NetworkNational Cancer Institute (NCI)CompletedRecurrent Adult Burkitt Lymphoma | Recurrent Adult Acute Lymphoblastic Leukemia | B-cell Adult Acute Lymphoblastic Leukemia | Acute Leukemias of Ambiguous Lineage | Philadelphia Chromosome Positive Adult Precursor Acute Lymphoblastic LeukemiaUnited States
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University of UtahLeadiant Biosciences, Inc.; Families of Spinal Muscular AtrophyCompletedSpinal Muscular Atrophy Type IUnited States, Canada, Germany
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Jazz PharmaceuticalsRecruitingTuberous Sclerosis Complex | Dravet Syndrome | Lennox Gastaut SyndromeUnited States
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Karunesh GangulyNational Institute on Deafness and Other Communication Disorders (NIDCD)RecruitingStroke | Multiple Sclerosis | Muscular Dystrophies | ALS | SCI - Spinal Cord InjuryUnited States
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Spruce BiosciencesActive, not recruitingCongenital Adrenal HyperplasiaUnited States, Australia, Brazil, Canada, Denmark, Estonia, Germany, Ireland, Italy, Korea, Republic of, Latvia, Lithuania, Netherlands, Poland, Romania, Spain, Sweden, Switzerland, Turkey, United Kingdom
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Progenity, Inc.CompletedDown Syndrome | Aneuploidy | DiGeorge Syndrome | Turner Syndrome | Klinefelter Syndrome | Chromosome Deletion | Edwards Syndrome | Patau SyndromeUnited States
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Sanguine BiosciencesCompletedMultiple Sclerosis | Parkinson's Disease | Amyotrophic Lateral Sclerosis (ALS) | Alzheimer's Disease | Huntington's DiseaseUnited States
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Spruce BiosciencesCompletedCongenital Adrenal Hyperplasia | CAH - Congenital Adrenal Hyperplasia | CAH - 21-Hydroxylase DeficiencyUnited States
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Roger PackerWithdrawnNeurofibromatosis Type 1 | Tumors of the Central Nervous SystemUnited States
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SanofiPulse Infoframe Ltd.RecruitingNiemann-Pick Diseases | Acid Sphingomyelinase DeficiencyUnited States
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Brigham and Women's HospitalUniversity of MichiganCompletedPrimary Aldosteronism | Primary Aldosteronism Due to Adrenal Hyperplasia (Bilateral)United States
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Mayo ClinicNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedNiemann-Pick Disease, Type CUnited States
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Eunice Kennedy Shriver National Institute of Child...Washington University School of Medicine; University of Oxford; National Human...CompletedNiemann-Pick Disease, Type CUnited States
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Eunice Kennedy Shriver National Institute of Child...TerminatedNiemann-Pick Disease, Type C1United States
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Vtesse, LLC, a Mallinckrodt Pharmaceuticals CompanyCompletedNiemann-Pick Disease, Type C1United States
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Cyclo Therapeutics, Inc.Active, not recruitingNiemann-Pick Disease, Type C1United States
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University of Texas Southwestern Medical CenterEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsSuspendedCongenital Adrenal HyperplasiaUnited States
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Eunice Kennedy Shriver National Institute of Child...RecruitingNiemann-Pick Disease, Type CUnited States
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Eunice Kennedy Shriver National Institute of Child...RecruitingNiemann-Pick Disease, Type CUnited States
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National Niemann-Pick Disease FoundationIcahn School of Medicine at Mount Sinai; Engage Health Inc.CompletedNiemann-Pick DiseasesUnited States
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Eunice Kennedy Shriver National Institute of Child...Washington University School of Medicine; Weill Medical College of Cornell...CompletedNeimann-Pick DiseaseUnited States
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Genzyme, a Sanofi CompanyTerminatedNiemann-Pick Disease | Acid Sphingomyelinase DeficiencyUnited States
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Masonic Cancer Center, University of MinnesotaRecruitingHurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Metachromatic Leukodystrophy | Alpha-Mannosidosis | Hunter Syndrome | Mucopolysaccharidosis Disorders | Maroteaux Lamy Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Glycoprotein Metabolic Disorders | Recessive Leukodystrophies | Globoid... and other conditionsUnited States
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University of California, San FranciscoNational Institute on Aging (NIA)CompletedDepression | Stress, Psychological | Dementia | Dementia, Vascular | Loneliness | Self Efficacy | Dementia, Mixed | Health Care Utilization | Dementia Alzheimers | Dementia of Alzheimer Type | Social Isolation | Dementia, Multi-Infarct | Dementia, Lewy Body | Dementia in Parkinsons Disease | Dementia Frontal | Dementia... and other conditionsUnited States
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Children's Hospital of PhiladelphiaIlera HealthcareTerminatedEpilepsy | Multiple Sclerosis | Glaucoma | Cancer | Parkinson Disease | HIV/AIDS | ALS | Autism Spectrum Disorder | Sickle Cell Disease | Spasticity, Muscle | Huntington Disease | Opioid Use | Neuropathy | Terminal Illness | Inflammatory Bowel Disease (IBD) | Intractable Pain | Ptsd | Dyskinetic SyndromeUnited States
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University of WashingtonEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsCompletedMultiple Sclerosis | Low Back Pain | Chronic Pain | Muscular Dystrophies | Spinal Cord Injury | Other and Unspecified Amputation Stump ComplicationsUnited States
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Washington University School of MedicineActive, not recruitingSpinal Muscular Atrophy | Spinal Muscular Atrophy Type 3 | Spinal Muscular Atrophy Type IIUnited States, Canada
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Adrenas Therapeutics IncRecruitingCongenital Adrenal HyperplasiaUnited States
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RTI InternationalEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationPrimary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditionsUnited States
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Zarife SahenkRecruitingCharcot-Marie-Tooth Disease | Peripheral Neuropathy | Chronic Inflammatory Demyelinating PolyneuropathyUnited States
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Talaris Therapeutics Inc.Duke UniversityNo longer availableMetachromatic LeukodystrophyUnited States
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Cyclo Therapeutics, Inc.CompletedNiemann-Pick Disease, Type C1United States
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Eunice Kennedy Shriver National Institute of Child...RecruitingCongenital Adrenal Hyperplasia (CAH) | Familial Male-Limited Precocious Puberty (FMPP)United States
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University of SienaRecruitingBehcet Syndrome | Uveitis | Hereditary Autoinflammatory Diseases | Schnitzler Syndrome | Scleritis | PFAPA Syndrome | Still Disease | Autoinflammatory Syndrome, Unspecified | Vexas SyndromeEgypt, Spain, Turkey, Italy, Saudi Arabia, Greece, Belgium, United States, Algeria, Brazil, Germany, Ghana, Iran, Islamic Republic of, Lebanon, Morocco, Poland, Portugal, Romania, Taiwan