A Coordinated Attack: Rett Syndrome Therapeutic Development. (April 2019)

• Record Type: Journal Article
• Authors: Gogliotti, Rocco G.; Niswender, Colleen M.
• Is Part Of: Trends in pharmacological sciences. Volume 40:Number 4(2019)
• Page Start: 233
• Page End: 236
• Publication Date: 2019-04
• Subjects: Rett syndrome -- pharmacological intervention; Pharmacology -- Periodicals; Pharmacology -- trends -- Periodicals; Pharmacologie -- Périodiques; Pharmacology; Electronic journals; Periodicals; 615.1
• DOI: 10.1016/j.tips.2019.02.007
• Languages: English
• ISSNs: 0165-6147

Abstract

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.