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Clinical Trials on Hypophosphatemic Rickets, X-Linked Dominant
Total 1552 results
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JCR Pharmaceuticals Co., Ltd.Enrolling by invitationMucopolysaccharidosis IIUnited States
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Rocket Pharmaceuticals Inc.Active, not recruitingDanon DiseaseUnited States
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JCR Pharmaceuticals Co., Ltd.Completed
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REGENXBIO Inc.CompletedMucopolysaccharidosis IIBrazil, United States, United Kingdom
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REGENXBIO Inc.Enrolling by invitation
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National Human Genome Research Institute (NHGRI)CompletedAutism | Asperger's Syndrome | Rett's DisorderUnited States
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ArmaGen, IncCompletedMucopolysaccharidosis IIUnited States, Germany, Philippines
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Soma JyonouchiBaylor College of MedicineCompletedX-linked Thrombocytopenia | Wiskott-Aldrich Syndrome (WAS)United States
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Ramathibodi HospitalAstraZenecaCompletedSpinobulbar Muscular Atrophy | Kennedy's DiseaseThailand
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National Institute of Neurological Disorders and...CompletedSpinal and Bulbar Muscular Atrophy | Kennedy's DiseaseUnited States
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University of Mississippi Medical CenterCompletedAutistic Disorder | Rett Syndrome | Asperger Syndrome | Pervasive Developmental Disorder | Childhood Disintegrative DisorderUnited States
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Janssen Pharmaceutical K.K.RecruitingX-Linked Retinitis PigmentosaJapan
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Frontera TherapeuticsPeking Union Medical College Hospital; Eye & ENT Hospital of Fudan University; Henan Provincial People's HospitalRecruitingX-Linked Retinitis PigmentosaChina
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Janssen Pharmaceutical K.K.RecruitingX-Linked Retinitis PigmentosaJapan
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Rocket Pharmaceuticals Inc.RecruitingDanon DiseaseUnited States
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University of ManchesterManchester University NHS Foundation Trust; Great Ormond Street Hospital for... and other collaboratorsRecruiting
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GC Biopharma CorpRecruitingMucopolysaccharidosis II | Hunter SyndromeKorea, Republic of
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Denali Therapeutics Inc.RecruitingMucopolysaccharidosis IIUnited States, Spain, United Kingdom, Czechia, France, Canada, Italy, Belgium, Netherlands, Germany, Argentina, Sweden, Turkey, Colombia
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Homology Medicines, IncWithdrawnMucopolysaccharidosis IIUnited States, Canada
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JCR Pharmaceuticals Co., Ltd.Active, not recruitingMucopolysaccharidosis IIBrazil
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ShireCompletedMucopolysaccharidosis IIUnited States, United Kingdom, Brazil, Germany
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AO GENERIUMCompletedA Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy VolunteersMetabolic Diseases | Mucopolysaccharidosis Type IIRussian Federation
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Hacettepe UniversityCompletedChewing Problem | Swallowing DisorderTurkey
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GlaxoSmithKlineCompletedMucopolysaccharidosis IIJapan
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Green Cross CorporationUnknownMucopolysaccharidosis II
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National Center for Research Resources (NCRR)University of California, San DiegoCompleted
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Janssen Research & Development, LLCJanssen Research & Development, LLCActive, not recruitingX-Linked Retinitis PigmentosaUnited States, Canada, Israel, United Kingdom, Spain, Denmark, France, Belgium, Italy, Netherlands, Switzerland
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BiogenCompletedX-Linked Retinitis PigmentosaUnited States, United Kingdom
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4D Molecular TherapeuticsActive, not recruitingX-Linked Retinitis PigmentosaUnited States
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NightstaRx Ltd, a Biogen CompanyCompletedChoroideremiaFinland, United States, Canada, Denmark, Netherlands, Germany, United Kingdom, Brazil, France
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Applied Genetic Technologies CorpCompletedX-Linked Retinitis PigmentosaUnited States
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Beacon TherapeuticsActive, not recruitingX-Linked Retinitis PigmentosaUnited States
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Beacon TherapeuticsEnrolling by invitationX-Linked Retinitis PigmentosaUnited States
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Beacon TherapeuticsActive, not recruitingX-Linked Retinitis PigmentosaUnited States
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TakedaRecruiting
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TakedaAvailableHunter SyndromeUnited States, Australia, Mexico, Spain, United Kingdom
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TakedaCompletedMucopolysaccharidosis (MPS)Brazil
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Green Cross CorporationCompleted
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Janssen Research & Development, LLCJanssen Research & Development, LLCActive, not recruitingX-Linked Retinitis PigmentosaBelgium, Canada, United States, Israel, United Kingdom, Spain, Denmark, France, Italy, Netherlands, Switzerland
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University Hospital, MontpellierAssociation Xtraordinaire sub-group DDX3X; GenidaUnknownIntellectual Developmental Disorder | X-LINKEDFrance
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Beacon TherapeuticsRecruitingX-Linked Retinitis PigmentosaUnited States
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Spark TherapeuticsCompletedChoroideremia | CHM (Choroideremia) Gene MutationsUnited States
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Uskudar UniversityRecruitingLesch-Nyhan SyndromeTurkey
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ShireCompletedHunter SyndromeUnited States, Spain, Canada, United Kingdom, Mexico, Australia, France
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Green Cross CorporationCompleted
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Lundquist Institute for Biomedical Innovation at...Active, not recruitingMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VIUnited States
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ShireCompleted
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Green Cross CorporationUnknownThe Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) PatientsHunter SyndromeKorea, Republic of
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SwanBio Therapeutics, Inc.RecruitingAMN | AMN Gene Mutation | X-ALDNetherlands, United States
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Eunice Kennedy Shriver National Institute of Child...TerminatedFragile X Syndrome | Premature Ovarian Failure | Premature Menopause | FMR1 Premutation | Primary Ovairan InsufficiencyUnited States