The phase 2A trial has its detractors despite positive animal results and is being sponsored by a company that is struggling financially.

The U.S. Food and Drug Administration plans to make an approval decision on the first-ever drug for girls and women with Rett syndrome by 12 March.

This month’s newsletter takes a close look at the orphan drug program in the United States, several cannabis-based therapies and a secondary analysis of bumetanide, among other new developments in autism-related drug trials.

FMR1 loss impairs sodium channels, hindering mouse neurons from generating the electrical signals needed to transmit information.

Going on Trial rounds up new developments in autism-related drug trials. This month we’re revisiting decade-old data from a trial of arbaclofen for fragile X syndrome and looking into a new implant-based approach to quelling seizures, among other treatment strategies.

Cells from people with fragile X syndrome overproduce — but don’t accumulate — proteins. New work suggests that excessive protein breakdown may account for this discrepancy, and explain some of the syndrome’s traits.

Many autism-linked genes are somehow tied to cilia, the tiny hair-like sensors that stud a cell’s surface. But the question remains whether, and how, cilia differences contribute to the condition.

A range of presentations at Neuroscience 2022 tie atypical social behavior to trouble discriminating between odors in the animals.

Scott describes his work on sound processing differences in the brains of zebrafish that model fragile X syndrome.

Data from two separate research teams suggest the cells are key to sensory hypersensitivity in fragile X syndrome.