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A Coordinated Attack: Rett Syndrome Therapeutic Development.

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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… Click to show full 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.

Keywords: coordinated attack; syndrome therapeutic; attack rett; rett syndrome; therapeutic development

Journal Title: Trends in pharmacological sciences
Year Published: 2019

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