A Coordinated Attack: Rett Syndrome Therapeutic Development.
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ABSTRACT:
SUBMITTER: Gogliotti RG
PROVIDER: S-EPMC6482467 | biostudies-literature | 2019 Apr
REPOSITORIES: biostudies-literature
Publications
A Coordinated Attack: Rett Syndrome Therapeutic Development.
Gogliotti Rocco G RG Niswender Colleen M CM
Trends in pharmacological sciences 20190401 4
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. ...[more]
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