Top sponsors: Assistance Publique - Hôpitaux de Paris (18) · National Human Genome Research Institute (NHGRI) (14) · Assiut University (12) · Mayo Clinic (11) · IRCCS Azienda Ospedaliero-Universitaria di Bologna (9)

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Active trials

• Brain Diseases, Metabolic, Inborn 346
• Lipid Metabolism, Inborn Errors 264
• Lysosomal Storage Diseases 262
• Carbohydrate Metabolism, Inborn Errors 195
• Amino Acid Metabolism, Inborn Errors 115
• Amyloidosis, Familial 108
• Purine-Pyrimidine Metabolism, Inborn Errors 77
• Metal Metabolism, Inborn Errors 76
• Steroid Metabolism, Inborn Errors 29
• Peroxisomal Disorders 28
• Renal Tubular Transport, Inborn Errors 28
• Adenine phosphoribosyltransferase deficiency 7

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• Hyperbilirubinemia, Hereditary 5
• Oculocerebrorenal Syndrome 4
• Drug Metabolism, Poor, CYP2D6-Related 3
• Progeria 3

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Data updated daily from ClinicalTrials.gov.