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Drug Repurposing for Treatment of NGLY1 Deficiency

TECHNOLOGY SUMMARY A class of FDA approved drugs may be repurposed for use in treating NGLY1 deficiency. NGLY1 deficiency is a rare genetic disorder, caused by a lack of the enzyme N-glycanase, characterized by a variety of debilitating and isolating symptoms, including global developmental delay, movement disorders, seizures, ocular abnormalities,...

Published: 6/6/2019 | Inventor(s): Kuberan (kuby) Balagurunathan, Matthew Brendon Might, Yiling Bi

Keywords(s):

Category(s): Therapeutics, Drug Repurposing