Cannabidiol (CBD) blocks the action of a molecule that drives an overexcitability feedback loop in a rodent model of epilepsy.

The approach improves the function of SYNGAP1-deficient neurons in vitro, but whether it will work in people remains unclear.

The drug, welcomed by patients, might be just the first of many.

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.

Time is running out to expose this disreputable push for profit over care and change insurance, funding and training practices for the better.

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.

After a year of intense growth, funding for biotech is in decline. The result is layoffs and program cuts — and maybe some innovation.

Early treatment with nutritional supplements and a high-protein diet forestalls some neurodevelopmental problems for children with BCKDK deficiency.

Both human and mouse progenitor cells with the alterations struggle to become neurons and instead express genes that are typically active only in muscle or the heart.

The treatment eases the animals’ sleep troubles, suggesting it has clinically meaningful effects beyond what was thought to be a critical window in early life.