The development of drugs for autism spectrum disorder (ASD) is hampered by the limited understanding of its pathophysiology, the heterogeneity of its symptoms, a dearth of experimental models, and the lack of experience in clinical development. In this Review, Ghosh and colleagues present recent insights into the molecular underpinnings of ASD and how these have translated into new tools for drug development, and also new approaches to overcome the particular challenges in this field.
- Anirvan Ghosh
- Aubin Michalon
- Luca Santarelli
