Clinical Trials on Genetic Diseases, Inborn in United States
Total 147152 results
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Ritter Pharmaceuticals, Inc.CompletedLactose IntoleranceUnited States
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Lysosomal and Rare Disorders Research and Treatment...UnknownLysosomal Storage DiseasesUnited States
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Brigham and Women's HospitalEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsRecruitingGenetic Predisposition to Disease | Hereditary DiseasesUnited States
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Mayo ClinicCompletedHemochromatosisUnited States
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University of Southern CaliforniaNational PKU AllianceActive, not recruitingPhenylketonuriasUnited States
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Emory UniversityBioMarin Pharmaceutical; Atlanta Clinical and Translational Science InstituteCompletedPhenylketonuriaUnited States
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University of Alabama at BirminghamVanderbilt University Medical CenterRecruiting
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National Human Genome Research Institute (NHGRI)Completed
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Apyx MedicalActive, not recruiting
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University of Texas Southwestern Medical CenterCompleted
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Ulthera, IncCompleted
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Ulthera, IncCompleted
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Ulthera, IncCompleted
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Ulthera, IncCompletedSkin LaxityUnited States
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Tulane UniversityAllerganCompleted
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Lysosomal and Rare Disorders Research and Treatment...SanofiUnknown
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Boston Children's HospitalWithdrawn
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National Institute of Neurological Disorders and...Completed
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Eunice Kennedy Shriver National Institute of Child...National Institutes of Health Clinical Center (CC); National Institute of Neurological... and other collaboratorsActive, not recruitingGenetic DisorderUnited States
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jasvinder singhNovartis; Takeda; Regeneron Pharmaceuticals; Birmingham, Alabama VA Medical CenterWithdrawn
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University of South FloridaCompleted
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Indiana UniversityCompletedLactose IntoleranceUnited States
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Stanford UniversityCompleted
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Applied Therapeutics, Inc.Active, not recruiting
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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Emory UniversityNational PKU AllianceCompleted
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Horizon Pharma Ireland, Ltd., Dublin IrelandCompletedUrea Cycle DisordersUnited States
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St. Jude Children's Research HospitalRecruiting
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Jerry Vockley, MD, PhDUltragenyx Pharmaceutical IncRecruitingMedium-chain Acyl-CoA Dehydrogenase DeficiencyUnited States
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Boston UniversityActive, not recruiting
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University of Texas Southwestern Medical CenterCompleted
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Cynosure, Inc.CompletedSkin LaxityUnited States
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Venus ConceptCompleted
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Ulthera, IncCompleted
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Ulthera, IncCompleted
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Ulthera, IncTerminatedSkin LaxityUnited States
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Taysha Gene Therapies, Inc.Withdrawn
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National Heart, Lung, and Blood Institute (NHLBI)CompletedHeart Diseases | Metabolism, Inborn ErrorsUnited States
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Children's Hospital Medical Center, CincinnatiSanofiRecruitingFabry DiseaseUnited States
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National Institute of Neurological Disorders and...CompletedFabry DiseaseUnited States
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National Institute of Neurological Disorders and...Completed
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National Institute of Neurological Disorders and...CompletedHealthy | Fabry DiseaseUnited States
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Oregon Health and Science UniversityNational Heart, Lung, and Blood Institute (NHLBI)TerminatedSmith-Lemli-Opitz SyndromeUnited States
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Horizon Therapeutics, LLCTerminatedUrea Cycle DisorderUnited States
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4D Molecular TherapeuticsActive, not recruitingChoroideremiaUnited States
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Brigham and Women's HospitalActive, not recruitingASCVD | Hyperlipemia, MixedUnited States
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National Human Genome Research Institute (NHGRI)RecruitingInborn Errors of Metabolism | Methylmalonic Acidemia | Organic AcidemiaUnited States