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A Coordinated Attack: Rett Syndrome Therapeutic Development

Authors

Gogliotti RG Rocco G , Niswender CM Colleen M .
Trends in pharmacological sciences. 2019 4 1; 40(4).
233-236

Abstract

Rett syndrome (RTT) is a neurodevelopmental disorder caused by mutations in the Methyl CpG binding protein 2 (MeCP2) gene. This Science & Society article focuses on pharmacological strategies that attack RTT treatment from multiple angles, including drug repurposing and de novo discovery efforts, and discusses the impacts of preclinical study design and translationally relevant outcome measures.

Tags: 2019