While Autism Spectrum Disorders (ASD) affect approximately one in 68 children, this hasn’t provoked much enthusiasm among pharmaceutical companies to develop drugs. However, recent advances in basic research Autism and novel gene-editing technologies may boost ASD drug development.
The reasons for low interest are a poor understanding of the biological mechanisms underlying Autism Spectrum Disorder, the multiple symptoms that make ASD difficult to diagnose and a shortage of animal models for the disease.
ASD is a general term for a wide spectrum of neurological disorders that share a general pattern of symptoms: repetitive behaviours, abnormal social interactions and impaired cognitive functions. These characteristics are frequently accompanied by epilepsy and intellectual disability, amongst others.
The severity of the symptoms arises from a combination of an individual’s genetic background and environmental factors. Many ASD disorders, such as the rare Fragile X syndrome, are passed on genetically, while others originate from novel genetic mutations that arise in only one family member.