Clinical Trials on X-linked Hypophosphatemic Rickets/Osteomalacia
Total 1579 results
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Jirair Krikor BedoyanUltragenyx Pharmaceutical IncRecruitingPyruvate Dehydrogenase Complex DeficiencyUnited States
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Cyprium Therapeutics, Inc.Enrolling by invitation
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University of FloridaEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsActive, not recruitingPyruvate Dehydrogenase Complex DeficiencyUnited States
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Fondazione TelethonCompletedPyruvate Dehydrogenase Complex DeficiencyItaly
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Baylor College of MedicineUniversity of California, San Diego; University of Alabama at Birmingham; Boston... and other collaboratorsCompletedPrader-Willi Syndrome | Rett Syndrome | Angelman Syndrome | Sleep ProblemsUnited States
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Eunice Kennedy Shriver National Institute of Child...Completed
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Center for Eye Research AustraliaCompleted
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ShireCovance; PRA Health Sciences; PharmaNetCompletedMucopolysaccharidosis II | Hunter Syndrome | MPS IIBrazil, Poland, Taiwan
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University of California, San DiegoBioMarin PharmaceuticalWithdrawnBehavioral Manifestations of Lesch-Nyhan DiseaseUnited States
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Lundquist Institute for Biomedical Innovation at...CompletedMucopolysaccharidosis Type I | Mucopolysaccharidosis Type II | Mucopolysaccharidosis Type VIUnited States
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Bio Products LaboratoryCompletedPrimary Immunodeficiency | Wiskott-Aldrich Syndrome | Hypogammaglobulinemia | Common Variable Hypogammaglobulinemia | X-linked Hypogammaglobulinemia | Immunodeficiency With Hyper-IgMUnited States
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Centre Hospitalier Universitaire de Saint EtienneCompletedBipolar Affective DisorderFrance
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TakedaRecruitingHunter SyndromeCanada, France
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Emalex Biosciences Inc.Psyadon PharmaTerminatedLesch-Nyhan Disease | Self-injurious BehaviorSpain, United States
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CENTOGENE GmbH RostockTerminatedMucopolysaccharidosis II | Hunter Syndrome | Hunter's Syndrome, Mild Form | Hunter's Canal SyndromeMexico
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Oslo University HospitalActive, not recruiting
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National Institute of Neurological Disorders and...CompletedMotor Neuron Disease | Spinal and Bulbar Muscular Atrophy (SBMA)United States
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National MPS SocietyMediResource Inc.TerminatedMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidoses | Mucopolysaccharidosis VI | Mucopolysaccharidosis IVUnited States
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Minoryx Therapeutics, S.L.Active, not recruitingAdrenoleukodystrophySpain, United States, Hungary, France, Germany, Italy, Netherlands, United Kingdom
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National Taiwan University HospitalCompleted
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Masonic Cancer Center, University of MinnesotaWithdrawn
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MedDay Pharmaceuticals SACompletedAdrenoleukodystrophy | Adrenomyeloneuropathy | AMNFrance, Germany, Spain
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Eunice Kennedy Shriver National Institute of Child...Food and Drug Administration (FDA)TerminatedAdrenoleukodystrophy | AdrenomyeloneuropathyUnited States
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Washington University School of MedicineRecruitingSevere Combined Immunodeficiency | Chronic Granulomatous Disease | DiGeorge Syndrome | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | X-linked Lymphoproliferative Syndrome | X-linked Agammaglobulinemia | Chediak-Higashi Syndrome | IPEX | Autoimmune Lymphoproliferative Syndrome | Common Variable... and other conditionsUnited States
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BiogenCompleted
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Lev-Hasharon Mental Healtlh CenterUnknownBipolar Affective DisorderIsrael
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Masonic Cancer Center, University of MinnesotaRecruiting
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Minoryx Therapeutics, S.L.RecruitingCerebral Adrenoleukodystrophy (cALD)United States, Brazil, Argentina
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Poxel SANot yet recruiting
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University of PittsburghNational Institute of Neurological Disorders and Stroke (NINDS); Rare Diseases...RecruitingPyruvate Dehydrogenase Complex Deficiency DiseaseUnited States
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bluebird bioCompletedCerebral Adrenoleukodystrophy (CALD)United States, France, Germany, Italy, Netherlands, United Kingdom
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Manchester University NHS Foundation TrustCompletedMucopolysaccharidosis Type IIUnited Kingdom
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Kunming Hope of Health HospitalTerminatedMenkes SyndromeChina
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bluebird bioCompletedCerebral Adrenoleukodystrophy (CALD)United States, Germany, Argentina, Australia, France, United Kingdom
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ShireCompletedHunter SyndromeSpain, United States, Canada, United Kingdom, France, Australia, Mexico
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Assistance Publique - Hôpitaux de ParisCentre National de la Recherche Scientifique, FranceCompletedAmyotrophic Lateral Sclerosis | Primary Lateral Sclerosis | Kennedy's DiseaseFrance
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University Hospital HeidelbergCompletedMucopolysaccharidosis Type I | Mucopolysaccharidosis Type II | Coping Behavior | Mucopolysaccharidosis Type III | Behavior DisordersGermany
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Masonic Cancer Center, University of MinnesotaWithdrawnCerebral Adrenoleukodystrophy
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FDA Office of Orphan Products DevelopmentHugo W. Moser Research Institute at Kennedy Krieger, Inc.Completed
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Far Eastern Memorial HospitalNational Taiwan University Hospital; Fu Jen Catholic UniversityUnknownAdrenoleukodystrophyTaiwan
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National Center for Research Resources (NCRR)Ohio State UniversityCompletedAmyotrophic Lateral Sclerosis | Spinal Muscular Atrophy | Spinobulbar Muscular Atrophy
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Stanford UniversityNational Heart, Lung, and Blood Institute (NHLBI); University of California... and other collaboratorsCompletedMuscular Dystrophy, DuchenneUnited States
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The Chaim Sheba Medical CenterRecruitingFragile X Syndrome | Williams Syndrome | Velocardiofacial SyndromeIsrael
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Kevin FlaniganMuscular Dystrophy AssociationTerminated
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BioLeaders CorporationCompleted
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Summit TherapeuticsCompletedModified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced DietMuscular Dystrophy, DuchenneUnited Kingdom
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bluebird bioCenter for International Blood and Marrow Transplant ResearchRecruitingCerebral Adrenoleukodystrophy (CALD)United States
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Poxel SANot yet recruiting