Roche’s gene therapy drug Rugonersen boosts expression of the protein missing in the syndrome in mice and monkeys, but whether it works in people remains to be seen.

Interim results from the previously paused trial suggest that doses of the experimental gene therapy drug GTX-102 are well tolerated in children with the autism-linked condition.

This month, we pore over null results from a study of the accuracy of emotion recognition skills in autistic people, clinical trials of a vasopressin drug called balovaptan, and an analysis of ‘systemizing’ abilities in autistic children.

People who have ‘profound autism’ — those with severe intellectual disability, limited communication abilities or both — tend to be excluded from research. The Autism Science Foundation seeks to change that.

After the vasopressin antagonist balovaptan flopped in a late-stage clinical trial, Suma Jacob and her colleagues took stock of all the factors that might have complicated the results.

Genetic factors could help explain why the ‘social hormone’ failed to benefit autistic people in a phase 3 clinical trial last year.

Swiss biotech Stalicla hopes to bring precision medicine to autism. Experts praise efforts to identify autism subgroups, but evidence to support the company’s claims has yet to be seen.

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.

In this edition, a strategy to help autistic children adapt their skills to new situations shows no benefit, but an early-life autism biomarker does.

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