Strategies to replace or compensate for mutated copies of the TCF4 gene could lead to treatments for this profound form of autism, a new study suggests.

A deactivated form of the gene editor restores UBE3A expression in mice and human neurons without cutting the genome. It may hold promise for future Angelman gene therapies.

Ramping up levels of one isoform of the autism-linked protein reverses traits in model mice, a new study shows.

The experimental compound, called GSK-LSD1, enhances social preferences and reduces repetitive grooming in mice, according to a new study.

Early interventions for autism lack solid data. The source of this problem is murky but may stem from ongoing debates about evidence quality and entrenched conflicts of interest within the field.

The ability to conduct large-scale screening in human neurons could accelerate the discovery of autism treatments.

Without it, autistic adults with high support needs like my son are missing out on treatments that could reduce their suffering and improve their quality of life.

Targeting the molecule, 4EPS, with an experimental drug may be a way to ease anxiety for autistic people, the researchers say. But not everyone is convinced.

The Rett Syndrome Global Registry is gathering data from people around the world with the rare condition to support clinical trials.

Girls and young women with Rett syndrome have less severe anxiety and breathing problems after treatment with trofinetide, an analogue of insulin-like growth factor 1, according to an unpublished placebo-controlled trial.