The development of drugs for autism spectrum disorder is hampered by the limited understanding of its pathophysiology, the heterogeneity of its symptoms and a dearth of experimental models. In this report, Ghosh and colleagues present recent insights into the molecular pathways involved in autism spectrum disorder and how these have translated into new tools for drug development. They propose a conceptual framework for drug discovery in autism spectrum disorder encompassing target identification, drug profiling and considerations for clinical trials in this novel area.
Ghosh A, Michalon A, Lindemann L, Fontoura P, Santarelli L: Drug discovery for autism spectrum disorder: challenges and opportunities. Nature Reviews Drug Discovery 12: 777–790 (2013) [Published online 01 October 2013; doi:10.1038/nrd4102 ].