Search clinical trials for: Natural History Study
Total 120839 results
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Institut de Myologie, FranceAssociation Française contre les Myopathies (AFM), ParisNot yet recruitingMerosin Deficient Congenital Muscular DystrophyFrance
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University Hospital TuebingenRecruitingRetinitis PigmentosaGermany
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National Taiwan University HospitalRecruiting
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National Institute of Nursing Research (NINR)Not yet recruitingRyanodine Receptor 1-Related Myopathy | Ryanodine Receptor 1 Related DisordersUnited States
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University College, LondonUniversity of North Carolina, Chapel Hill; NYU Grossman School of MedicineRecruitingDentatorubral-Pallidoluysian AtrophyUnited States, United Kingdom
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Centre Hospitalier Régional de la CitadelleRecruitingAngelman SyndromeBelgium
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University of PennsylvaniaRecruitingLCAT DeficiencyUnited States
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Rocket Pharmaceuticals Inc.Recruiting
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Atamyo TherapeuticsNot yet recruiting
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McGill University Health Centre/Research Institute...Canadian Institutes of Health Research (CIHR)RecruitingZellweger Spectrum DisorderCanada
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Radboud University Medical CenterUMC UtrechtNot yet recruitingCentronuclear Myopathy | Nemaline Myopathy | Central Core Disease | Multi-Minicore DiseaseNetherlands
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Elpida Therapeutics SPCNot yet recruitingNeuromuscular Diseases | Peripheral Neuropathy | Neuro-Degenerative Disease
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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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Radboud University Medical CenterRecruitingSELENON-related Myopathy | LAMA2-related Muscular DystrophyNetherlands
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Puerta de Hierro University HospitalParc Taulí Hospital UniversitariRecruiting
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National Human Genome Research Institute (NHGRI)RecruitingMetabolic Disease | Purine-Pyrimidine Metabolism | AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2 | UNG, OMIM *191525, Hyper-IgM Syndrome 5 | NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency | UMPS, OMIM *613891, Orotic Aciduria | DHODH, OMIM *126064,... and other conditionsUnited States
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Centre Hospitalier Universitaire de NiceRecruitingFacioscapulohumeral Muscular Dystrophy Type 2France, Italy, Spain
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argenxRecruitingCongenital Myasthenic SyndromeUnited States, Canada, France
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Regeneron PharmaceuticalsRecruitingCongenital Hearing Loss Secondary to Biallelic Mutations in the Otoferlin Gene (OTOF) | Biallelic Mutations in the Gap Junction Beta 2 (GJB2) Gene | Digenic Mutations in GJB2/Gap Junction Beta 6 (GJB6) GenesUnited States
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Friedreich's Ataxia Research AllianceRecruitingFriedreich AtaxiaUnited States, Canada, New Zealand, Australia, France, Austria, Belgium, Brazil, Czechia, Germany, Greece, Ireland, Italy, Spain, United Kingdom, India, Netherlands
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PfizerRecruitingCardiomyopathy, Dilated | Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM)United States, Spain, Netherlands, Poland, United Kingdom
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National Cancer Institute (NCI)RecruitingAcute Lymphoblastic Leukemia | Relapsed/Refractory | B CellUnited States
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Massachusetts General HospitalNorthwestern University; University of Florida; University of Minnesota; Henry... and other collaboratorsRecruitingALS | Kennedy Disease | MND (Motor Neurone DIsease) | PLS | PMA - Progressive Muscular Atrophy | PBP - Progressive Bulbar PalsyUnited States, Italy
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Tobias MoserRecruitingHearing Impairment | Otoferlin-related Auditory SynaptopathyGermany
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Johan VirhammarSwedish Society for Medical ResearchRecruitingHydrocephalus | Normal Pressure HydrocephalusSweden, Finland, Italy
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Hereditary Neuropathy FoundationRecruitingCharcot-Marie-Tooth Disease | Charcot-Marie-Tooth Disease, Type IA | Charcot-Marie-Tooth Disease Type 2A | Charcot-Marie-Tooth | Charcot-Marie-Tooth Disease, Type IB | Charcot-Marie-Tooth Disease Type 2 | Charcot-Marie-Tooth Disease, Type 2C | Charcot-Marie-Tooth Disease Type 2A2B | Charcot-Marie-Tooth... and other conditionsUnited States
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University of California, San DiegoNew York University; Heinrich-Heine University, Duesseldorf; Universität TübingenRecruitingGenetic Disease | Mitochondrial Encephalomyopathies | Neonatal Encephalopathy | Mutation | Hereditary Spastic Paraplegia | White Matter Disease | Spastic ParaplegiaUnited States
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Phoenix NestAparito Ltd.; Vaincres Les Maladies Lysosomales; Association Sanfilippo SudNot yet recruitingSanfilippo Syndrome Type CUnited States, France
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Columbia UniversityNational Eye Institute (NEI); Centre Hospitalier National d'Ophtalmologie des... and other collaboratorsRecruitingRetinitis Pigmentosa | Best Vitelliform Macular DystrophyUnited States, Germany, France
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Complement TherapeuticsRecruitingEye Diseases | Macular Degeneration | Retinal Degeneration | Geographic Atrophy | Dry Age-related Macular Degeneration | Retinal Disease | Macular AtrophyUnited Kingdom, United States
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Massachusetts General HospitalRecruiting
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Tel-Aviv Sourasky Medical CenterRecruitingCreutzfeldt-Jakob Disease (CJD)Israel
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Eunice Kennedy Shriver National Institute of Child...RecruitingLymphatic Diseases | Lymphatic AbnormalitiesUnited States
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National Heart, Lung, and Blood Institute (NHLBI)RecruitingMantle Cell Lymphoma | Chronic Lymphocytic Leukemia (CLL) | Sudden Cardiac Death | Hematologic Malignancies | Cardiac Arrhythmias | Waldenstr(SqrRoot)(Delta)m s MacroglobulinemiaUnited States
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Lysosomal and Rare Disorders Research and Treatment...Team SanfilippoRecruitingSanfilippo Syndrome | MPS3United States
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Asklepios Biopharmaceutical, Inc.RecruitingParkinson's Disease | Multiple System Atrophy | Multiple System Atrophy, Parkinson VariantUnited States, Poland
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National Cancer Institute (NCI)RecruitingFanconi Anemia | Inherited Bone Marrow Failure SyndromeUnited States
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National Institute of Mental Health (NIMH)RecruitingMigraine | Bipolar Disorder | Major DepressionUnited States
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National Institute of Environmental Health Sciences...Not yet recruiting
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National Human Genome Research Institute (NHGRI)RecruitingGenetic ConditionsUnited States
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National Institute of Allergy and Infectious Diseases...RecruitingPregnancy | Healthy Controls | Pediatric Illnesses | Inflammatory DiseasesUnited States
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National Institute of Allergy and Infectious Diseases...RecruitingObstructive Sleep Apnea | Sleep Disordered Breathing | Tonsillitis | Periodic Fever, Aphthous Stomatitis, Pharyngitis, And Cervical Adenitis (Pfapa) | Tonsil DisorderUnited States
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National Institute of Diabetes and Digestive and...RecruitingChronic Hepatitis B Virus (Hbv)United States
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Jaeb Center for Health ResearchFoundation Fighting BlindnessRecruitingRetinitis Pigmentosa | Inherited Retinal DegenerationUnited States, United Kingdom, France, Brazil, Canada, Finland, Israel, Mexico, Netherlands, Switzerland, Australia
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Columbia UniversityRecruitingImmune Deficiency | Allergic Inflammation | Immune Dysregulation DisorderUnited States
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Johns Hopkins UniversityNeurofibromatosis Therapeutic Acceleration ProgramRecruitingNeurofibromatosis 1 | Neurofibromatosis Type 1 | Cutaneous Neurofibroma | Neurofibromatosis (Nonmalignant)United States
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Children's Hospital Medical Center, CincinnatiCerebral Palsy AllianceRecruiting
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Akouos, Inc.Eli Lilly and CompanyRecruitingSensorineural Hearing Loss, BilateralUnited States, Taiwan, Germany, Spain, Australia, United Kingdom
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LMU KlinikumUniversity of Pisa; German Federal Ministry of Education and Research; European...RecruitingMitochondrial Diseases | MDS | Mitochondrial Myopathies | MELAS Syndrome | MIDD | Kearns-Sayre Syndrome | MERRF Syndrome | Barth Syndrome | Leigh Syndrome | MNGIE | LHON | Pearson Syndrome | NARP Syndrome | Coenzyme Q10 Deficiency | SANDO | SCAE | MIRAS | CPEOAustria, Germany, Italy
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Eunice Kennedy Shriver National Institute of Child...Not yet recruitingCardiovascular Phenotype | Metabolic Phenotype | Hypertensive Disease | Psychological Phenotype | AudioUnited States