Clinical Trials on Hypophosphatemic Rickets, X-Linked Dominant
Total 1562 results
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Merve KurtRecruitingDuchenne Muscular Dystrophy | Virtual Reality | Spinal Muscular Atrophy | Neuromuscular Disease | BiofeedbackTurkey
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Yonsei UniversityRecruitingFabry DiseaseKorea, Republic of
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Yonsei UniversityRecruitingFabry's DiseaseKorea, Republic of
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Genzyme, a Sanofi CompanyTerminatedFabry Disease | Alpha Galactosidase A DeficiencyUnited States, Austria, United Kingdom
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Columbia UniversityMuscular Dystrophy Association; Stevens Institute of TechnologyCompletedDuchenne Muscular Dystrophy | Spinal Muscular Atrophy Type 3United States
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Resverlogix CorpWithdrawn
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University Children's Hospital, ZurichCompletedPhenylketonurias | OTC Deficiency | Inborn Errors of Metabolism | Urea Cycle Disorder | Maple Syrup Urine Disease | Methylmalonicacidemia | Aminoacidopathy | Patient Reported Outcome MeasurementsSwitzerland
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University of Maryland, BaltimoreCompletedOsteoporosis PseudogliomaUnited States
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Ulla Feldt-RasmussenCompleted
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Wolfson Medical CenterUnknownFabry Disease in the Young StrokeIsrael
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Genzyme, a Sanofi CompanyWithdrawn
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National Institute of Neurological Disorders and...CompletedHealthy | Fabry Disease | Cerebrovascular AccidentUnited States
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Assistance Publique - Hôpitaux de ParisNot yet recruitingHemophilia A | Hemophilia B | Cystic Fibrosis | Sickle Cell Disease | Muscular Dystrophy, Duchenne | Fragile X Syndrome | Huntington Disease | Myotonic Dystrophy | Autosomal Recessive Polycystic Kidney Disease | Neurofibromatosis-Noonan Syndrome | Muscular Dystrophy, Becker | Invasive PreNatal Diagnosis in a... and other conditionsFrance
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Lysosomal and Rare Disorders Research and Treatment...Recruiting
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Chiesi Farmaceutici S.p.A.Active, not recruitingFabry DiseaseUnited States, Netherlands, Australia, Hungary, United Kingdom, Canada, Czechia, Finland, France, Italy, Norway, Slovenia, Spain
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University of Maryland, BaltimoreChildren's Hospital of PhiladelphiaWithdrawnOsteoporosis Pseudoglioma SyndromeUnited States
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CENTOGENE GmbH RostockWithdrawnStroke, Acute | Cerebrovascular Accident | Cerebral StrokeGermany
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Children's Hospital Medical Center, CincinnatiCompletedDuchenne Muscular Dystrophy | Spinal Muscular Atrophy | Congenital Muscular Dystrophy
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Rennes University HospitalCompleted
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University College, LondonUniversity of Sydney; University Hospital BirminghamUnknownFabry DiseaseUnited Kingdom, Australia
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Assistance Publique - Hôpitaux de ParisTerminatedNeuromuscular Diseases | Amyotrophic Lateral Sclerosis | Duchenne Muscular DystrophyFrance
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Genzyme, a Sanofi CompanyCompletedFabry DiseaseUnited States, United Kingdom, Canada, Hungary, Poland, Czech Republic
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National Institute of Neurological Disorders and...CompletedFabry DiseaseUnited States
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Centre Hospitalier Universitaire de LiegeSanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaboratorsRecruitingCongenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditionsBelgium
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iECURE, Inc.RecruitingUrea Cycle Disorders, Inborn | Ornithine Transcarbamylase Deficiency | Ornithine Transcarbamylase Deficiency Disease | Ornithine Carbamoyltransferase Deficiency (Disorder)United Kingdom
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Yonsei UniversityRecruitingFabry DiseaseKorea, Republic of
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ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseUnited States, Netherlands, Hungary, United Kingdom, Czechia, Norway, Slovenia, Spain, Finland, France, Italy, Switzerland
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Mads Peter Godtfeldt StemmerikEdgewise Therapeutics, Inc.CompletedBecker Muscular Dystrophy | McArdle Disease | Limb-Girdle Muscular Dystrophy Type 2Denmark
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NYU Langone HealthNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)CompletedCystinuria | Primary Hyperoxaluria | Dent Disease | Adenine Phosphoribosyl Transferase DeficiencyUnited States, Iceland
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Novo Nordisk A/SCompletedHealthy | Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors | Acquired Bleeding Disorder | Acquired Haemophilia | Congenital FVII Deficiency | Glanzmann's DiseaseFrance
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University of CambridgeSanofiRecruiting
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Fundacion para la Investigacion Biomedica del Hospital...Fundación Mutua MadrileñaRecruiting
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Baylor College of MedicineChildren's Hospital of Philadelphia; Children's Hospital Colorado; Seattle Children... and other collaboratorsRecruitingOrnithine Transcarbamylase Deficiency | Urea Cycle Disorder | Argininosuccinic Aciduria | Hyperargininemia | Citrullinemia 1 | ARGI Deficiency | ASL Deficiency | ASS DeficiencyUnited States
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University Hospital, AngersRecruiting
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Chiesi Farmaceutici S.p.A.Active, not recruitingFabry DiseaseBelgium, United States, United Kingdom, Italy, Czechia, Denmark, Norway
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Assistance Publique - Hôpitaux de ParisShire International GmbHCompleted
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National Human Genome Research Institute (NHGRI)Completed
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University Hospital, BordeauxCompletedPain | Fabry's DiseaseFrance
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Masonic Cancer Center, University of MinnesotaRecruitingCommon Variable Immunodeficiency | Chronic Granulomatous Disease | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | X-linked Lymphoproliferative Disease | Langerhan's Cell Histiocytosis | Chediak-Higashi Syndrome | Griscelli Syndrome | Bare Lymphocyte Syndrome | SCID | Omenn's Syndrome | Reticular... and other conditionsUnited States
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University of MichiganNational Institute of Dental and Craniofacial Research (NIDCR)Not yet recruitingHealing Wound | Alveolar Bone Loss | Image | Dent Disease | Alveolar; Wound
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SanofiRecruitingFabry DiseaseChina, Finland, Germany, Norway, United Kingdom, United States, Denmark, Austria, Greece, Romania, Argentina, Brazil, Canada, Japan, Mexico, Poland, Italy, France, Turkey, Australia, Switzerland
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Albina Nowak, MDSwiss National Science FoundationRecruiting
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Assistance Publique - Hôpitaux de ParisCompletedDuchenne Muscular Dystrophy | Spinal Muscular AtrophyFrance
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Turkish Society of Pediatric Gastroenterology,...CompletedDuchenne Muscular Dystrophy | Pompe Disease (Late-onset)Turkey
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Universidad de Zaragozafundación española para el estudio y tratamiento de la Enfermedad de Gaucher... and other collaboratorsCompleted
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Boston Children's HospitalTerminatedFanconi Anemia | Dyskeratosis CongenitaUnited States
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Amicus TherapeuticsCompleted
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University of Alabama at BirminghamCompleted
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University of Alabama at BirminghamCompletedProteinuria | Fabry DiseaseUnited States, Slovenia