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A member asked:
Angelman syndrome, what are the latest developments to treat this?
A doctor has provided 1 answer
Topoisomerase drugs: Angelman syndrome has a variety of neurological and physical issues which are treated supportively. However, the disease is caused by silencing of the ube3a gene. Topoisomerase inhibitors like topotecan and Etoposide have been used in an animal model to unsilenced the ube3a gene in brain neurons. This class of drug may be useful for early treatment.
Answered 6/24/2015
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