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Total 2692 results
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Memorial Sloan Kettering Cancer CenterRecruitingRefractory Acute Lymphoblastic Leukemia | Philadelphia-Negative ALL | Philadelphia-Positive ALL | Refractory Acute Lymphoblastic Leukemia (ALL) | Relapsed ALL, Adult | Refractory Acute Lymphoid Leukemia in RelapseUnited States
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InnoVec Biotherapeutics Inc.Peking Union Medical College HospitalRecruitingEye Diseases | Retinal Degeneration | Retinal Disease | RetinoschisisChina
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Specila hospital for cerebral palsy and developmental...FRAXA Research FoundationRecruitingFragile X SyndromeSerbia
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BioMarin PharmaceuticalRecruitingDuchenne Muscular DystrophySpain, United Kingdom
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Perha PharmaceuticalsGöteborg University; Proteas BioanalyticsRecruiting
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University Hospital, ToulouseNot yet recruitingPrader-Willi SyndromeFrance
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UniQure Biopharma B.V.RecruitingFabry DiseaseUnited States
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Sarepta Therapeutics, Inc.RecruitingDuchenne Muscular DystrophyUnited States
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Université de SherbrookeCanadian Institutes of Health Research (CIHR); Centre de recherche du Centre... and other collaboratorsNot yet recruiting
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Université de SherbrookeCanadian Institutes of Health Research (CIHR); Centre de recherche du Centre... and other collaboratorsNot yet recruitingFragile X Syndrome
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Brunel UniversityRoyal Free Hospital NHS Foundation TrustRecruiting
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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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Hospices Civils de LyonNot yet recruitingX-Linked Hypophosphatemic Rickets
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Riphah International UniversityRecruiting
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Centre Hospitalier Universitaire de LiegeCentre Hospitalier Régional de la Citadelle; SYSNAVRecruitingAngelman SyndromeBelgium
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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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University of CambridgeSanofiRecruiting
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Sardocor Corp.Not yet recruitingDMD-Associated Dilated Cardiomyopathy
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bluebird bioCenter for International Blood and Marrow Transplant ResearchRecruitingCerebral Adrenoleukodystrophy (CALD)United States
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Nanfang Hospital, Southern Medical UniversityRecruitingAcute Lymphoblastic Leukemia | Philadelphia Chromosome | Adult ALL | Philadelphia-Positive ALL | IKZF1 Gene MutationChina
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Boston University Charles River CampusBoston Children's HospitalNot yet recruiting
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Rocket Pharmaceuticals Inc.Recruiting
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Richard FryeAutism Discovery and Treatment FoundationRecruitingEpilepsy | Neurodevelopmental Disorders | Autism Spectrum Disorder | Down Syndrome | Mitochondrial Encephalomyopathies | Pediatric Acute-Onset Neuropsychiatric Syndrome | Cerebral Folate Deficiency | Pediatric Autoimmune Neuropsychiatric Disorder Associated With Streptococcal InfectionUnited States
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Children's Hospital of Fudan UniversityNot yet recruiting
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Perha PharmaceuticalsFondation Jérôme Lejeune; European Innovation Council; France 2030 programRecruitingHealthy Volunteers | Down Syndrome | Alzheimer's DiseaseFrance
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University Hospital, Strasbourg, FranceRecruiting
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University College CorkNot yet recruitingGestational Diabetes | Neonatal Encephalopathy | Diastolic Dysfunction | Trisomy 21 | Transient Tachypnea of the Newborn | Small for Gestational Age at DeliveryIreland
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Children's Hospital of Fudan UniversityRecruiting
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Riphah International UniversityRecruiting
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Riphah International UniversityRecruitingDown SyndromePakistan
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University of SevilleRecruitingDown Syndrome | Physical Disability | FamilySpain
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Azienda Ospedaliero-Universitaria di ModenaRecruitingTurner Syndrome | Anomaly HeartItaly
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Minoryx Therapeutics, S.L.RecruitingX-linked AdrenoleukodystrophyFrance
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ACADIA Pharmaceuticals Inc.RecruitingHyperphagia in Prader-Willi SyndromeUnited States, Canada, United Kingdom
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Qilu Hospital of Shandong UniversityRecruitingFabry Disease | Hypertrophic Cardiomyopathy | Left Ventricular HypertrophyChina
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Gianni SoraruMario Negri Institute for Pharmacological ResearchRecruitingSpinal and Bulbar Muscular AtrophyItaly
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National Institute for Mother and Child Health...Marie Curie Emergency Children's Hospital, Bucharest; Victor Gomoiu Children...RecruitingChronic Granulomatous DiseaseRomania
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Hospices Civils de LyonRecruiting
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Changhai HospitalNot yet recruiting
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Taysha Gene Therapies, Inc.RecruitingRett SyndromeUnited States, United Kingdom
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Ionis Pharmaceuticals, Inc.RecruitingPelizaeus-Merzbacher DiseaseFrance, Netherlands
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safia Darweesh halwshNot yet recruiting
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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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Mitsubishi Tanabe Pharma America Inc.RecruitingErythropoietic Protoporphyria (EPP) | X-Linked Protoporphyria (XLP)United States, Spain, France, Japan, United Kingdom, Italy, Australia, Poland, Bulgaria, Czechia, Netherlands
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Foundation for Prader-Willi ResearchRTI InternationalRecruitingPrader-Willi SyndromeUnited States
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Tianjin Central Hospital of Gynecology ObstetricsRecruitingChromosome AberrationsChina
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Solid Biosciences Inc.RecruitingDuchenne Muscular DystrophyUnited States
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Riphah International UniversityRecruiting
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Hoffmann-La RocheSarepta Therapeutics, Inc.Recruiting
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National Cancer Institute (NCI)Not yet recruiting