Top sponsors: Assistance Publique - Hôpitaux de Paris (79) · National Cancer Institute (NCI) (48) · National Institute of Allergy and Infectious Diseases (NIAID) (43) · Assiut University (39) · Sanofi (35)

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Active trials

• Metabolism, Inborn Errors 888
• Heredodegenerative Disorders, Nervous System 724
• Skin Diseases, Genetic 549
• Genetic Diseases, X-Linked 444
• Hemoglobinopathies 384
• Anemia, Hemolytic, Congenital 378
• Eye Diseases, Hereditary 375
• Neoplastic Syndromes, Hereditary 339
• Muscular Dystrophies 254
• Chromosome Disorders 241
• Blood Coagulation Disorders, Inherited 238
• Cystic Fibrosis 203

Show all 45

• Primary Immunodeficiency Diseases 131
• Ciliopathies 127
• Osteochondrodysplasias 96
• Laminopathies 86
• Dwarfism 83
• Hereditary Autoinflammatory Diseases 71
• Imprinting Disorders 44
• alpha 1-Antitrypsin Deficiency 34
• Adrenal Hyperplasia, Congenital 29
• Anemia, Hypoplastic, Congenital 29
• Renal Tubular Transport, Inborn Errors 28
• Osteogenesis Imperfecta 26
• CADASIL 19
• Lennox Gastaut Syndrome 17
• Marfan Syndrome 16
• Brugada Syndrome 15
• Camurati-Engelmann Syndrome 12
• Alagille Syndrome 11
• Ataxia Telangiectasia 9
• GATA2 Deficiency 9
• Myasthenic Syndromes, Congenital 7
• Platelet Glycoprotein IV Deficiency 7
• Autoimmune Lymphoproliferative Syndrome 6
• Cerebral Palsy, Ataxic, Autosomal Recessive 5
• Costello Syndrome 5
• Hyper-IgM Immunodeficiency Syndrome 5
• Kartagener Syndrome 5
• Loeys-Dietz Syndrome 5
• Kallmann Syndrome 4
• Oculocerebrorenal Syndrome 4
• Cardiomyopathy, Hypertrophic, Familial 3
• CHARGE Syndrome 3
• Cryoglobulinemia, Familial Mixed 3

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Data updated daily from ClinicalTrials.gov.