Clinical Trials on Autosomal or Sex Linked Recessive Genetic Disease
Total 14135 results
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Murdoch Childrens Research InstituteUniversity of Sydney; Sydney Children's Hospitals Network; Women's and Children... and other collaboratorsActive, not recruitingAutosomal Recessive Disorder | X-Linked Genetic DiseasesAustralia
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University of GuelphMcMaster UniversityRecruitingAutosomal Recessive Disorder (Genetic Carriers of PKU)Canada
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University of GuelphMcMaster University; Laval UniversityNot yet recruitingAutosomal Recessive Disorder (Genetic Carriers of PKU)Canada
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Otsuka Pharmaceutical Development & Commercialization...RecruitingAutosomal Recessive Polycystic Kidney (ARPKD)United States, United Kingdom, Belgium, Poland, France, Germany, Italy
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Kadmon Corporation, LLCCompletedPolycystic Kidney, Autosomal RecessiveUnited States
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Otsuka Pharmaceutical Development & Commercialization...RecruitingAutosomal Recessive Polycystic Kidney Disease (ARPKD)United States, Belgium, Spain, Germany, United Kingdom, Poland
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National Heart, Lung, and Blood Institute (NHLBI)CompletedParkinson Disease 6, Early-Onset | Parkinson Disease (Autosomal Recessive, Early Onset) 7, Human | Parkinson Disease Autosomal Recessive, Early Onset | Parkinson Disease, Autosomal Recessive Early-Onset, Digenic, Pink1/Dj1United States
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National Institute of Allergy and Infectious Diseases...Enrolling by invitationChronic Granulomatous Disease | p47 | Autosomal RecessiveUnited States
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Xiaofan ZhuAvailableFanconi Anemia | Autosomal or Sex Linked Recessive Genetic Disease | Bone Marrow Hematopoiesis Failure, Multiple Congenital Abnormalities, and Susceptibility to Neoplastic Diseases. | Hematopoiesis Maintainance.China
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Newcastle-upon-Tyne Hospitals NHS TrustRecruitingBethlem Myopathy | Ullrich Congenital Muscular Dystrophy 1, Digenic, Col6A1/Col6A2 | Ullrich Congenital Muscular Dystrophy 1, Autosomal Recessive | Ullrich Congenital Muscular Dystrophy 1, Autosomal Dominant | Bethlem Myopathy 1, Autosomal Recessive | UCMD | BTHLM1United Kingdom
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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI); National Heart, Lung, and Blood Institute...CompletedSevere Combined Immunodeficiency | Adenosine Deaminase Deficiency | Immune System Disorder | Autosomal Recessive Disorder | Purine-Nucleoside Phosphorylase Deficiency | Severe Combined Immunodeficiency With Absence of T and B Cells | X-Linked Severe Combined ImmunodeficiencyUnited States
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CSL BehringCompletedCommon Variable Immunodeficiency | X-linked Agammaglobulinemia | Autosomal Recessive AgammaglobulinemiaGermany, United Kingdom, France, Italy, Poland, Romania, Spain, Sweden, Switzerland
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Krystal Biotech, Inc.Not yet recruitingAutosomal Recessive IchthyosisUnited States
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Origin BiosciencesCompletedMolybdenum Cofactor Deficiency (MoCD) | Rare Autosomal Recessive Disorder | Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)United States
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Krystal Biotech, Inc.Active, not recruitingTGM-1 Related Autosomal Recessive Congenital IchthyosisUnited States
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Alkeus Pharmaceuticals, Inc.RecruitingStargardt Disease | Stargardt Macular Degeneration | Stargardt Macular Dystrophy | Autosomal Recessive Stargardt Disease 1 (ABCA4-related)United States
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Alkeus Pharmaceuticals, Inc.Enrolling by invitationStargardt Disease | Stargardt Macular Degeneration | Stargardt Macular Dystrophy | Autosomal Recessive Stargardt Disease 1 (ABCA4-related)United States
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Assistance Publique - Hôpitaux de ParisNot yet recruitingAutosomal Recessive Congenital Ichthyosis | Epidermolytic IchthyosisFrance
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University of California, DavisCompletedFragile X Syndrome | Genetic DiseasesUnited States
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Assistance Publique - Hôpitaux de ParisCompletedSpinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia, Autosomal Recessive 3 | Episodic Ataxia, Type 7France
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Children's Hospital Medical Center, CincinnatiEunice Kennedy Shriver National Institute of Child Health and Human Development...CompletedFragile X SyndromeUnited States
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Seaside Therapeutics, Inc.CompletedFragile X SyndromeUnited States
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Seaside Therapeutics, Inc.National Institutes of Health (NIH)CompletedFragile X SyndromeUnited States
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Children's Hospital Medical Center, CincinnatiRecruitingFragile X SyndromeUnited States
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Esoterix Genetic Laboratories, LLCUnknownFragile X SyndromeUnited States
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IRCCS Fondazione Stella MarisRecruitingFragile X SyndromeItaly
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Specila hospital for cerebral palsy and developmental...FRAXA Research FoundationRecruitingFragile X SyndromeSerbia
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Children's Hospital Medical Center, CincinnatiEunice Kennedy Shriver National Institute of Child Health and Human Development...RecruitingFragile X SyndromeUnited States
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Tetra Discovery PartnersRecruitingFragile X SyndromeUnited States
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Zynerba Pharmaceuticals, Inc.RecruitingFragile X SyndromeUnited States, Australia, United Kingdom, Ireland
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National Institute of Mental Health (NIMH)CompletedHealthy Volunteers | Fragile X SyndromeUnited States
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Zynerba Pharmaceuticals, Inc.Enrolling by invitationFragile X SyndromeUnited States, United Kingdom, New Zealand, Australia
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Elizabeth Berry-KravisNational Institute of Neurological Disorders and Stroke (NINDS); Novartis PharmaceuticalsCompleted
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Zynerba Pharmaceuticals, Inc.CompletedFragile X SyndromeUnited States, New Zealand, Australia
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Children's Hospital Medical Center, CincinnatiRush University Medical CenterCompleted
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Novartis PharmaceuticalsCompletedFragile X SyndromeUnited States, Switzerland, Germany, Australia, Canada, Denmark, France, Italy, Spain, United Kingdom
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Stanford UniversityCompletedFragile X SyndromeUnited States
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Parc de Salut MarCompleted
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University of California, DavisCompleted
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Université de SherbrookeFRAXA Research FoundationCompletedFragile X SyndromeCanada
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University of California, DavisThe National Fragile X FoundationCompleted
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Alcobra Ltd.CompletedFragile X SyndromeUnited States, Israel
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Novartis PharmaceuticalsTerminatedFragile X SyndromeGermany, United States, Switzerland, Australia, Canada, Denmark, France, Italy, United Kingdom, Spain
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Seaside Therapeutics, Inc.Completed
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Stanford UniversityCompleted
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Ludwig-Maximilians - University of MunichCompletedInclusion Body Myositis, Sporadic | Inclusion Body Myopathy, Autosomal-recessive | Inclusion Body Myopathy, Autosomal-dominant | Congenital Cataracts, Facial Dysmorphism, And NeuropathyGermany
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Massachusetts General HospitalNational Institutes of Health (NIH)UnknownHealthy | Fragile X SyndromeUnited States
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Craig EricksonHealx LimitedRecruitingFragile X SyndromeUnited States
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Elizabeth Berry-KravisCompletedFragile X SyndromeUnited States
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Novartis PharmaceuticalsCompletedFragile X SyndromeUnited States, Spain