A Coordinated Attack: Rett Syndrome Therapeutic Development

Publication date: April 2019Source: Trends in Pharmacological Sciences, Volume 40, Issue 4Author(s): Rocco G. Gogliotti, Colleen M. NiswenderRett 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.
Source: Trends in Pharmacological Sciences - Category: Drugs & Pharmacology Source Type: research