Top sponsors: Sanofi (9) · Genzyme, a Sanofi Company (8) · Amicus Therapeutics (7) · Assiut University (7) · Assistance Publique - Hôpitaux de Paris (6)

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Active trials

• Lysosomal Storage Diseases, Nervous System 182
• Phenylketonurias 45
• Hereditary Central Nervous System Demyelinating Diseases 38
• Hepatolenticular Degeneration 35
• Urea Cycle Disorders, Inborn 30
• Adrenoleukodystrophy 19
• Leigh Disease 9
• MELAS Syndrome 9
• Homocystinuria 6
• Creatine deficiency, X-linked 5
• Pyruvate Dehydrogenase Complex Deficiency Disease 5
• Refsum Disease 5

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• Zellweger Syndrome 5
• Oculocerebrorenal Syndrome 4
• Cerebral Amyloid Angiopathy, Familial 3
• Galactosemias 3
• Menkes Kinky Hair Syndrome 3

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Data updated daily from ClinicalTrials.gov.