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Search clinical trials for: genetic disorders
Total 154527 results
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Taysha Gene Therapies, Inc.RecruitingRett SyndromeUnited States, United Kingdom
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Amir MoheetRecruitingCystic Fibrosis | Cystic Fibrosis-related DiabetesUnited States
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NYU Langone HealthNot yet recruitingFamilial DysautonomiaUnited States
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Perminder SachdevMelbourne Health; Royal Brisbane and Women's Hospital; The University of Queensland and other collaboratorsRecruiting
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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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TESS Research FoundationBrown University; Stanford University; University of Texas Southwestern Medical...RecruitingEpilepsy | Movement Disorders | Rare Diseases | Genetic Disorder | Citrate Transporter Deficiency | SLC13A5 Deficiency | EIEE25 | Kohlschutter-Tonz Syndrome (Non-ROGDI) | Citrate Transporter Disorder | DEE25United States
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Kiminori Kimura, MDJapan Agency for Medical Research and DevelopmentRecruiting
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Foundation for Prader-Willi ResearchRTI InternationalRecruitingPrader-Willi SyndromeUnited States
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IRCCS San RaffaeleNot yet recruiting
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Jiangsu Gensciences lnc.RecruitingSevere Hemophilia AChina
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Jiangsu Gensciences lnc.Not yet recruitingSevere Hemophilia AChina
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University Hospital, Clermont-FerrandNot yet recruiting
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PYC TherapeuticsNot yet recruitingOptic Atrophies, Hereditary | Autosomal Dominant Optic Atrophy | Optic Atrophy, Autosomal Dominant | Kjer Optic AtrophyAustralia
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Assistance Publique - Hôpitaux de ParisRecruiting
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Solid Biosciences Inc.RecruitingDuchenne Muscular DystrophyUnited States
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Arrowhead PharmaceuticalsRecruitingMyotonic Dystrophy 1New Zealand, Australia
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Azienda Ospedaliero-Universitaria di ModenaRecruitingIron Overload | Hemochromatosis | Thalassemia Major | Thalassemia Intermedia | EndocrinopathyItaly
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Radboud University Medical CenterRecruitingSELENON-related Myopathy | LAMA2-related Muscular DystrophyNetherlands
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Massachusetts General HospitalJohns Hopkins UniversityRecruitingNeurofibromatosis 1United States
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Arrowhead PharmaceuticalsRecruitingFacio-Scapulo-Humeral DystrophyNew Zealand
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McMaster UniversityNot yet recruitingObesity | Lysosomal Storage Diseases | Muscle Loss | Glycogen Storage Disease Type II | Pompe Disease | Nutrition Poor | Glycogen Storage Disease Type II, Adult | Glycogen Storage Disease Type II Late Onset
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Riphah International UniversityRecruiting
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NYU Langone HealthNot yet recruiting
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Intellia TherapeuticsRegeneron PharmaceuticalsRecruitingTransthyretin Amyloidosis (ATTR) With CardiomyopathyUnited States, Australia, United Kingdom, New Zealand, Canada
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Hoffmann-La RocheSarepta Therapeutics, Inc.Recruiting
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Martin DichgansRecruitingCerebral Amyloid AngiopathyGermany
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First Affiliated Hospital Xi'an Jiaotong UniversityRecruitingHomozygous Familial HypercholesterolemiaChina
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National Cancer Institute (NCI)Not yet recruiting
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Fundació Sant Joan de DéuHospital Universitari Vall d'Hebron Research Institute; Fundació La Marató... and other collaboratorsRecruitingParenting | Genetic Syndrome | Language Development | Autism or Autistic TraitsSpain
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Affiliated Hospital of Guangdong Medical UniversityNot yet recruitingHemophilia B Without Inhibitor
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Vanderbilt University Medical CenterRecruitingDuchenne Muscular DystrophyUnited States
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BiocadRecruitingStudy of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)Hemophilia BRussian Federation
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Zhejiang UniversityNot yet recruitingHemophilia B Without Inhibitor
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Seagen Inc.RecruitingLymphoma, Large B-Cell, Diffuse | Lymphoma, Non-Hodgkin | Hodgkin Disease | Lymphoma, T-Cell, Cutaneous | Lymphoma, T-Cell, Peripheral | Lymphoma, Large-Cell, AnaplasticUnited States
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Affiliated Hospital of Nantong UniversityNot yet recruitingDepression | Polymerase Chain Reaction | Polymorphism, Genetic | CYP1A2 Polymorphism | Plasma Concentration | CYP2C9 Polymorphism
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Massachusetts General HospitalJohns Hopkins UniversityRecruitingNeurofibromatosis 1United States
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Amicus TherapeuticsRecruitingPompe DiseaseUnited States
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Motric BioRecruitingStroke | Multiple Sclerosis | Cerebral Palsy | Spinal Cord Injuries | Dystonia | Muscle Spasticity | Hereditary Spastic Paraplegia | Hypertonia, MuscleUnited States
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Université de Reims Champagne-ArdenneNot yet recruiting
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Lysosomal and Rare Disorders Research and Treatment...RecruitingBone Diseases | Gaucher DiseaseUnited States
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University Hospital, BordeauxNot yet recruitingMonogenic Obesity | Non Syndromic ObesityFrance
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West China HospitalRecruiting
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University College, LondonRecruitingHereditary Sensory Neuropathy Type IUnited Kingdom
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AceLink Therapeutics, Inc.Tigermed Consulting Co., LtdRecruiting
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Puerta de Hierro University HospitalParc Taulí Hospital UniversitariRecruiting
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Shanghai Belief-Delivery BioMed Co., LtdShanghai MYGT Biopharmaceutical LLCRecruiting
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Verve Therapeutics, Inc.Not yet recruitingHypercholesterolemia | Atherosclerotic Cardiovascular Disease | Heterozygous Familial HypercholesterolemiaNew Zealand, United Kingdom
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Hacettepe UniversityNot yet recruitingDuchenne Muscular DystrophyTurkey