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Clinical Trials on Genetic Disease, X-Linked
Total 15138 results
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STZ eyetrialUniversity Hospital TuebingenCompleted
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Children's Hospital Medical Center, CincinnatiNot yet recruitingAutism Spectrum Disorder | Fragile X Syndrome
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University Hospital, ToursCompletedHealthy Volunteers | Autism | Fragile-X Syndrome (FXS)France
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BiogenCompletedChoroideremiaUnited States, Finland, France, Denmark, Netherlands, Germany, United Kingdom, Canada
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Ariel UniversityASST Santi Paolo e CarloCompleted
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Great Ormond Street Hospital for Children NHS Foundation...WithdrawnGranulomatous Disease, Chronic, X-linked, VariantUnited Kingdom
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Indiana UniversityCompletedFragile X Syndrome | Autism Spectrum DisordersUnited States
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Eunice Kennedy Shriver National Institute of Child...University of WashingtonUnknownWiskott-Aldrich Syndrome | Leukocyte Adhesion Deficiency Syndrome | X-Linked Agammaglobulinemia | X-Linked Hyper IgM SyndromeUnited States
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Astellas Gene TherapiesCompletedX-linked Myotubular MyopathyUnited States, France, United Kingdom, Canada, Germany
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University of FloridaAudentes TherapeuticsCompletedX-linked Myotubular MyopathyUnited States
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University Hospital ErlangenPervormance GmbHCompletedShort Term Effects and Risks of Physical Exercise in Subjects With Hypohidrotic Ectodermal DysplasiaX-Linked Hypohidrotic Ectodermal Dysplasia | Exercise-induced Rise of Body TemperatureGermany
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Stanford UniversityTerminatedFragile X Syndrome (FXS) | Idiopathic Intellectual Developmental Disorder (IDD)United States
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Icahn School of Medicine at Mount SinaiNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Rare... and other collaboratorsCompletedErythropoietic Protoporphyria | EPP | XLP | X-Linked Protoporphyria | XLPP | X-Linked Dominant Erythropoietic Protoporphyria | XLEPP | XLDPUnited States
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Nova Mentis Life Science CorpKGK Science Inc.SuspendedCognitive Dysfunction | Fragile X Syndrome | BehaviorCanada
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RespireRxFRAXA Research FoundationCompletedFragile X Syndrome | AutismUnited States
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University of North Carolina, Chapel HillCompletedFragile X Syndrome | Psychosis | Autism Spectrum DisordersUnited States
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Ionis Pharmaceuticals, Inc.Not yet recruitingMethyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
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James DowlingCanadian Institutes of Health Research (CIHR); Cures Within Reach; The Joshua... and other collaboratorsRecruitingX Linked Myotubular MyopathyUnited Kingdom, Canada, United States
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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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Baylor College of MedicineCompleted
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bluebird bioTerminatedCerebral Adrenoleukodystrophy (CALD) | Adrenoleukodystrophy (ALD) | X-Linked Adrenoleukodystrophy (X-ALD)United States, United Kingdom, Argentina, Canada, Germany, Italy, Netherlands
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National Institute of Allergy and Infectious Diseases...CompletedImmunoproliferative DisorderUnited States
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Rare Thyroid Therapeutics International ABVitaccess LtdCompletedAllan-Herndon-Dudley Syndrome | Monocarboxylate Transporter 8 (MCT8) DeficiencyUnited Kingdom
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bluebird bioActive, not recruitingCerebral Adrenoleukodystrophy (CALD) | Adrenoleukodystrophy (ALD) | X-Linked Adrenoleukodystrophy (X-ALD)United States, Argentina, Australia, Brazil, France, Germany, Netherlands, United Kingdom, Italy
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State University of New York - Upstate Medical...TerminatedFamilial Encephalopathy With Neuroserpin Inclusion Bodies | X-linked Mental Retardation | Congenital Vertical Talus | Idiopathic Generalised Epilepsy | Familial DementiaUnited States
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Astellas Gene TherapiesCompletedMales With X-linked Myotubular Myopathy (XLMTM)United States, United Kingdom
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Beijing Tiantan HospitalRecruiting
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University of MinnesotaNo longer availableX-linked AdrenoleukodystrophyUnited States
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Academisch Medisch Centrum - Universiteit van Amsterdam...The Stop ALD FoundationCompletedAdrenomyeloneuropathy | X-linked AdrenoleukodystrophyNetherlands
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Children's Hospital Medical Center, CincinnatiEunice Kennedy Shriver National Institute of Child Health and Human Development...RecruitingAutism Spectrum Disorder | Autistic Disorder | Fragile X Syndrome | Asperger SyndromeUnited States
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NightstaRx Ltd, a Biogen CompanyCompletedChoroideremiaFinland, United States, Canada, Denmark, Netherlands, Germany, United Kingdom, Brazil, France
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Ionis Pharmaceuticals, Inc.RecruitingMethyl CpG Binding Protein 2 (MECP2) Duplication SyndromeUnited States
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Assistance Publique - Hôpitaux de ParisBraintaleNot yet recruitingX-linked Adrenoleukodystrophy
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Stanford UniversityNational Institute of Neurological Disorders and Stroke (NINDS); Hugo W. Moser... and other collaboratorsCompletedX-linked AdrenoleukodystrophyUnited States
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Shenzhen Geno-Immune Medical InstituteUnknown
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NeuroVia, Inc.WithdrawnX-Linked AdrenoleukodystrophyAustralia, Argentina, United Kingdom, Colombia, Chile, France, Russian Federation, Ukraine
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Rare Thyroid Therapeutics International ABPremier Research Group plc; Egetis TherapeuticsRecruitingMonocarboxylate Transporter 8 Deficiency | Allan-Herndon-Dudley SyndromeUnited States, United Kingdom, Netherlands
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Universitair Ziekenhuis BrusselMarguerite - Marie Delacroix FoundationNot yet recruitingClassical Lissencephalies and Subcortical Band HeterotopiasBelgium
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Rare Thyroid Therapeutics International ABEgetis Therapeutics; AnovoRxAvailableMonocarboxylate Transporter 8 Deficiency | Allan-Herndon-Dudley SyndromeUnited States
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National Eye Institute (NEI)CompletedRetinoschisis | X-LinkedUnited States
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Spark TherapeuticsCompletedChoroideremia | CHM (Choroideremia) Gene MutationsUnited States
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University of Alabama at BirminghamUniversity of Colorado, Denver; Eunice Kennedy Shriver National Institute of... and other collaboratorsCompletedRett Syndrome, Preserved Speech Variant | Mecp2 Duplication Syndrome | Rett-related DisordersUnited States
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Bambino Gesù Hospital and Research InstituteAutour Des Williams; AceaUnknownFragile X Syndrome | Williams SyndromeItaly
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Onofre, Aurora Pujol, M.D.Instituto de Salud Carlos III; Fundacion Hesperia; ELA España AssociationCompletedAdrenomyeloneuropathy | X-linked AdrenoleukodystrophySpain
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Hugo W. Moser Research Institute at Kennedy Krieger...European Leukodystrophy AssociationCompletedX-linked AdrenoleukodystrophyUnited States
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Rocket Pharmaceuticals Inc.Recruiting
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University of California, San DiegoRocket Pharmaceuticals Inc.Recruiting
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Cure CMDUniversity of Michigan; Congenital Muscle Disease International RegistrCompleted
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TakedaRecruitingSpinal and Bulbar Muscular AtrophyJapan