Top sponsors: JCR Pharmaceuticals Co., Ltd. (8) · Duke University (6) · Denali Therapeutics Inc. (5) · Genzyme, a Sanofi Company (5) · QED Therapeutics, a BridgeBio company (5)

Narrower conditions

Active trials

• Mucopolysaccharidoses 75
• Glycogen Storage Disease 65
• Pyruvate Metabolism, Inborn Errors 19
• Hyperoxaluria, Primary 16
• alpha-Mannosidosis 6
• Lactose Intolerance 6
• Mucolipidoses 5
• Sucrase-isomaltase deficiency, congenital 5
• Glucosephosphate Dehydrogenase Deficiency 4
• Congenital Disorders of Glycosylation 3
• Galactosemias 3
• Glut1 Deficiency Syndrome 3

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Data updated daily from ClinicalTrials.gov.