Angelman syndrome

news

First molecular therapeutic for Angelman syndrome to advance into clinical trials

23 March 2023 | By Ria Kakkad (Drug Target Review)

The scientists from Texas A&M have developed GTX-102, a novel therapeutic candidate to target Angelman syndrome by reactivating expression of deficient protein.

news

Gene therapy shows promise for Angelman syndrome treatment

26 October 2021 | By Anna Begley (Drug Target Review)

A new gene therapy restored motor skill-learning and usual behaviours in Angelman syndrome mouse models, suggesting a novel therapy for the condition.

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Angelman syndrome

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