Clinical Trials on Steroid Metabolism, Inborn Errors
Total 4320 results
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Oregon Health and Science UniversityNational Heart, Lung, and Blood Institute (NHLBI)TerminatedSmith-Lemli-Opitz SyndromeUnited States
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Neurogene Inc.RecruitingNeuronal Ceroid Lipofuscinosis CLN5United States, United Kingdom
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Polaryx Therapeutics, Inc.Not yet recruitingJuvenile Neuronal Ceroid Lipofuscinosis
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REGENXBIO Inc.Active, not recruitingNeuronal Ceroid Lipofuscinosis Type 2United Kingdom
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University of RochesterBatten Disease Support and Research Assocation (BDSRA)CompletedJuvenile Neuronal Ceroid LipofuscinosisUnited States
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StemCells, Inc.CompletedNeuronal Ceroid LipofuscinosisUnited States
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Eunice Kennedy Shriver National Institute of Child...RecruitingBatten Disease | Juvenile Neuronal Ceroid Lipofuscinosis (CLN3)United States
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Amicus TherapeuticsTerminatedBatten Disease | Neuronal Ceroid Lipofuscinosis CLN6 | Neuronal Ceroid Lipofuscinosis CLN3United States
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Amicus TherapeuticsCompletedVariant Late-Infantile Neuronal Ceroid LipofuscinosisUnited States
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BioMarin PharmaceuticalActive, not recruitingLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2United States
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Weill Medical College of Cornell UniversityNational Institute of Neurological Disorders and Stroke (NINDS); National Institutes... and other collaboratorsCompletedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States
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Weill Medical College of Cornell UniversityTerminatedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States
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IRCCS Fondazione Stella MarisCompletedNeuronal Ceroid-LipofuscinosesItaly
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Neurogene Inc.RecruitingCeroid Lipofuscinosis, Neuronal 5United States
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StemCells, Inc.WithdrawnNeuronal Ceroid LipofuscinosisUnited States
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Jessica ScherrBioMarin PharmaceuticalEnrolling by invitationBatten Disease | Neuronal Ceroid-Lipofuscinoses | CLN2United States
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Weill Medical College of Cornell UniversityNathan's Battle FoundationCompletedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States
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Weill Medical College of Cornell UniversityNational Institutes of Health (NIH)CompletedBatten Disease | Late-Infantile Neuronal Ceroid LipofuscinosisUnited States
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Weill Medical College of Cornell UniversityCompletedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States
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REGENXBIO Inc.WithdrawnNeuronal Ceroid Lipofuscinosis Type 2 (CLN2)
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Eunice Kennedy Shriver National Institute of Child...CompletedInfantile Neronal Ceroid LipofuscinosisUnited States
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REGENXBIO Inc.WithdrawnLate-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
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BioMarin PharmaceuticalCompletedBatten Disease | CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Jansky-Bielschowsky DiseaseUnited States, Italy, Germany, United Kingdom
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University of RochesterRecruitingBatten Disease | Neuronal Ceroid Lipofuscinosis | Neuronal Ceroid Lipofuscinosis CLN6 | Neuronal Ceroid Lipofuscinosis CLN3 | Neuronal Ceroid Lipofuscinosis CLN5 | Neuronal Ceroid Lipofuscinosis CLN1 | Neuronal Ceroid Lipofuscinosis CLN2 | Neuronal Ceroid Lipofuscinosis CLN7 | Neuronal Ceroid Lipofuscinosis... and other conditionsUnited States
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Emily de los ReyesRecruitingBatten Disease | CLN6United States
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Nationwide Children's HospitalMassachusetts General HospitalCompletedNeuronal Ceroid Lipofuscinosis | Batten's DiseaseUnited States
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BioMarin PharmaceuticalCompletedBatten Disease | CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Jansky-Bielschowsky Disease | CLN2 DisorderUnited States, Germany, Italy, United Kingdom
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BioMarin PharmaceuticalCompletedBatten Disease | CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Jansky-Bielschowsky Disease | CLN2 DisorderUnited States, Germany, Italy, United Kingdom
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Weill Medical College of Cornell UniversityNathan's Battle FoundationCompletedBatten Disease | Late-Infantile Neuronal Ceroid Lipfuscinosis
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David L Rogers, MDActive, not recruitingNeuronal Ceroid Lipofuscinosis Type 2United States
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Beyond Batten Disease FoundationTheranexusActive, not recruitingBatten DiseaseUnited States
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The Royal Wolverhampton Hospitals NHS TrustCompletedApparent Mineralocorticoid ExcessUnited Kingdom
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Amicus TherapeuticsActive, not recruiting
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Amicus TherapeuticsActive, not recruitingBatten Disease | CLN6United States
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Brigham and Women's HospitalCompletedApparent Mineralocorticoid Excess (AME)United States
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Eunice Kennedy Shriver National Institute of Child...RecruitingNiemann-Pick Disease Type C1 | Juvenile Neuronal Ceroid Lipofuscinosis | Smith-Lemli-Opitz Syndrome | Creatine Transporter DeficiencyUnited States
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Children's Hospital of Orange CountyActive, not recruitingNeuronal Ceroid-Lipofuscinoses | Neuronal Ceroid Lipofuscinosis CLN2 | Spinocerebellar Ataxia, Autosomal Recessive 7United States
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University of MiamiHRSA/Maternal and Child Health BureauCompletedPhenylketonuria | Inborn Errors of Metabolism | GalactosemiaUnited States
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Sciotec Diagnostic Technologies GmbHNot yet recruitingFructose IntoleranceAustria
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Augusta UniversityUniversity of Iowa; Teikyo UniversityRecruitingFructose IntoleranceUnited States
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Augusta UniversityUnknownFructose IntoleranceUnited States
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Mayo ClinicNational Institute of Neurological Disorders and Stroke (NINDS)Enrolling by invitationCongenital Disorders of GlycosylationUnited States
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Northwestern UniversityWithdrawnCHILD Syndrome | Smith Lemli Opitz Syndrome | Syndromic Ichthyoses | Conradi Syndrome
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Icahn School of Medicine at Mount SinaiEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsCompletedApparent Mineralocorticoid Excess SyndromeUnited States, Brazil, France
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University of LausanneCompletedGlucose Metabolism Disorders | Fructose Metabolism, Inborn Errors | Hereditary Fructose Intolerance
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Augusta UniversityTerminatedDietary Fructose IntoleranceUnited States
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University of LausanneUniversity of LiegeCompletedHereditary Fructose IntoleranceBelgium
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Maastricht University Medical CenterPfizerCompleted
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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