Clinical Trials on Bulbo-Spinal Atrophy, X-Linked
Total 1847 results
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National Institute of Allergy and Infectious Diseases...TerminatedGrowth Failure | X-linked Severe Combined Immunodeficiency (XSCID) | Growth Hormone ResistenceUnited States
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Marco CapogrossoRoche-GenentechNot yet recruitingSpinal Muscular Atrophy | Spinal Muscular Atrophy Type 3 | SMA | Spinal Muscular Atrophy Type II | Spinal Muscular Atrophy 4United States
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Edimer PharmaceuticalsCompletedX-linked Hypohidrotic Ectodermal DysplasiaUnited States
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Hospices Civils de LyonUnknownX-linked Mental RetardationFrance
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University of California, DavisUniversity of Alberta; St. Justine's HospitalRecruitingNeurobehavioral Manifestations | Genetic Diseases, X-Linked | Intellectual Disability | Fragile X Syndrome | Sex Chromosome Disorders | Fragile X Mental Retardation Syndrome | Trinucleotide Repeat Expansion | Fra(X) Syndrome | FXS | Mental Retardation, X LinkedUnited States, Canada
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Cure CMDUniversity of Michigan; Congenital Muscle Disease International RegistrCompleted
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Hoffmann-La RocheRecruitingSpinal Muscular Atrophy (SMA)Belgium, United States, Croatia, Japan, Netherlands, Spain, Canada, Poland, United Kingdom, Portugal, Italy, Australia
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Northwell HealthCompletedAdult Spinal Muscular AtrophyUnited States
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Hugh McMillanFamilies of Spinal Muscular Atrophy; Gwendolyn Strong FoundationTerminatedSpinal Muscular Atrophy (SMA)Canada
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National Taiwan University HospitalNational Science Council, TaiwanUnknownSpinal Muscular Atrophy | Spinal Cord Injury | MyelomeningoceleTaiwan
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Novartis Gene TherapiesActive, not recruitingSMA | Spinal Muscular Atrophy Type II | Spinal Muscular Atrophy Type I | Spinal Muscular Atrophy Type IIIUnited States, Belgium, France, Japan, United Kingdom, Italy, Taiwan, Australia, Canada
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Mitsubishi Tanabe Pharma America Inc.RecruitingErythropoietic Protoporphyria (EPP) | X-Linked Protoporphyria (XLP)United States, Spain, France, United Kingdom, Japan, Italy, Australia, Bulgaria, Czechia, Netherlands, Poland
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Edimer PharmaceuticalsCompletedX Linked Hypohidrotic Ectodermal DysplasiaUnited States
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University Hospital ErlangenEdimer PharmaceuticalsCompletedX-linked Hypohidrotic Ectodermal DysplasiaGermany
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AveXis, Inc.United BioSource, LLCRecruitingSpinal Muscular Atrophy (SMA)Japan, United States, Korea, Republic of, Israel, Greece, Ireland, Portugal, Russian Federation, Taiwan
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Catalyst Pharmaceuticals, Inc.TerminatedSpinal Muscular Atrophy Type 3Italy
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Novartis Gene TherapiesCompletedGene Therapy | SMA - Spinal Muscular AtrophyUnited States
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The Hong Kong Polytechnic UniversityThe University of Hong Kong; Manchester Metropolitan UniversityTerminatedSpinal Muscular Atrophy Type 3Hong Kong
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David WilliamsActive, not recruitingGene Therapy | Severe Combined Immunodeficiency, X LinkedUnited States, United Kingdom
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National Institute of Allergy and Infectious Diseases...UnknownX-Linked Combined Immunodeficiency DiseasesUnited States
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Edimer PharmaceuticalsActive, not recruitingX-linked Hypohidrotic Ectodermal DysplasiaUnited States, United Kingdom, France, Germany, Italy
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National Institute of Allergy and Infectious Diseases...CompletedSevere Combined ImmunodeficiencyUnited States
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CENTOGENE GmbH RostockCompletedScoliosis | Muscular Atrophy | Duchenne Muscular Dystrophy | Lordosis | Muscular Weakness | Increased Lordosis/Scoliosis | Hyporeflexia | Red-Green Color BlindnessEgypt, India, Albania, Pakistan, Georgia, Lebanon, Romania, Sri Lanka
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BiocadRecruitingSpinal Muscular Atrophy (SMA)Belarus, Russian Federation
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Ohio State UniversityMassachusetts General Hospital; National Institute of Neurological Disorders... and other collaboratorsCompletedSpinal Muscular Atrophy (SMA)United States
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Hoffmann-La RocheAssociation Française contre les Myopathies (AFM), ParisCompletedSpinal Muscular Atrophy Type II | Spinal Muscular Atrophy Type III Non AmbulantGermany, Italy, France, Belgium, Poland, Netherlands, United Kingdom
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EspeRare FoundationIqvia Pty Ltd; Pierre Fabre MedicamentRecruitingX-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)United States, Spain, France, Germany, Italy, United Kingdom
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National Institute of Allergy and Infectious Diseases...CompletedGenetic Diseases, X-Linked | Lymphoproliferative Disease | X-Linked Lymphoproliferative DiseaseUnited States
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Fondazione Policlinico Universitario Agostino Gemelli...CompletedUltrasonography | Spinal Muscular Atrophy | Anesthesia, Spinal | Spinal Puncture ComplicationsItaly
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Istanbul Medipol University HospitalIstanbul UniversityRecruitingNeuromuscular Diseases | Spinal Muscular Atrophy Type 3Turkey
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Washington University School of MedicineActive, not recruitingSpinal Muscular Atrophy | Spinal Muscular Atrophy Type 3 | Spinal Muscular Atrophy Type IIUnited States, Canada
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Scholar Rock, Inc.CompletedNeuromuscular Diseases | Neuromuscular Manifestations | Atrophy | Muscular Atrophy | Spinal Muscular Atrophy | Muscular Atrophy, Spinal | Spinal Muscular Atrophy Type 3 | SMA | Spinal Muscular Atrophy Type 2United States, Spain, Italy, Netherlands
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University Hospital, Basel, SwitzerlandEnrolling by invitationSpinal Muscular Atrophy Type II | Spinal Muscular Atrophy Type IIISwitzerland
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Assistance Publique - Hôpitaux de ParisCompleted
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WestatNational Institute of Neurological Disorders and Stroke (NINDS)TerminatedSpinal Muscular Atrophy Type II | Spinal Muscular Atrophy Type IIIUnited States
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BiogenRecruitingSpinal Muscular Atrophy (SMA)France
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Natera, Inc.WithdrawnGenetic Testing | Spinal Muscular Atrophy (SMA) | Carrier ScreeningUnited States
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BiogenNo longer availableInfantile-onset Spinal Muscular AtrophyNew Zealand, Colombia, Turkey
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Grand Valley State UniversityActive, not recruitingCerebral Palsy | Spinal Cord Injuries | Spinal Muscular Atrophy | Genetic Disease | Spina Bifida | Development DelayUnited States
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Children's Hospital of Chongqing Medical UniversityRecruiting
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Great Ormond Street Hospital for Children NHS Foundation...RecruitingSevere Combined Immunodeficiency, X-LinkedUnited Kingdom
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Scholar Rock, Inc.Active, not recruitingNeuromuscular Diseases | Neuromuscular Manifestations | Atrophy | Muscular Atrophy | Spinal Muscular Atrophy | Muscular Atrophy, Spinal | Spinal Muscular Atrophy Type 3 | SMA | Spinal Muscular Atrophy Type 2 | Anti-myostatinUnited States, Germany, Belgium, Spain, United Kingdom, Netherlands, France, Italy, Poland
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Nationwide Children's HospitalNovartisActive, not recruitingSpinal Muscular Atrophy Type II | Spinal Muscular Atrophy Type IUnited States
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Assistance Publique - Hôpitaux de ParisCompletedInfantile Spinal Muscular Atrophy of Type 2 or 3France
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The Hospital for Sick ChildrenCompletedX-linked Lymphoproliferative Syndrome Type 2 (XLP-2)Canada
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Novartis PharmaceuticalsRecruitingSpinal Muscular AtrophiesBrazil
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Scholar Rock, Inc.Active, not recruitingNeuromuscular Diseases | Neuromuscular Manifestations | Atrophy | Muscular Atrophy | Spinal Muscular Atrophy | Muscular Atrophy, Spinal | Spinal Muscular Atrophy Type 3 | SMA | Anti-myostatin | Spinal Muscular Atrophy Type IIUnited States, Spain, Belgium, Germany, Netherlands, Poland, Italy, France, United Kingdom
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Icahn School of Medicine at Mount SinaiRecruitingAutism Spectrum Disorder | DDX3X | Mental Retardation, X-linked 102United States
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Novartis PharmaceuticalsRecruitingSpinal Muscular Atrophy (SMA)Spain, Canada, Taiwan, Belgium, Japan, Italy, Singapore, United Kingdom, France, Australia, Denmark, United States
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Seoul National University HospitalRecruitingDuchenne Muscular Dystrophy | Scoliosis Neuromuscular | Lordosis LumbarKorea, Republic of