A Coordinated Attack: Rett Syndrome Therapeutic Development.

Gogliotti RG, Niswender CM
Trends Pharmacol Sci. 2019 40 (4): 233-236

PMID: 30905360 · PMCID: PMC6482467 · DOI:10.1016/j.tips.2019.02.007

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.

Copyright © 2019 Elsevier Ltd. All rights reserved.

MeSH Terms (10)

Animals Drug Discovery Drug Evaluation, Preclinical Drug Repositioning Female Humans Methyl-CpG-Binding Protein 2 Mutation Research Design Rett Syndrome

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