Despite grave side effects, it’s vital to keep developing treatments for rare genetic forms of childhood epilepsy, says West, president of the KCNT1 Epilepsy Foundation and father of a son with the condition.

The experienced clinician discusses writing his recent paper, and its reception in the field.

The new devices, which monitor neural activity from within blood vessels, show long-term stability in rats and could one day deliver electrical stimulation.

This month’s Going on Trial newsletter explores how organ-on-a-chip models could smooth the transition from preclinical to clinical trials, among other drug development news.

The newfound DNA-cutting enzyme, called Fanzor, can be programmed to edit the human genome and could prove easier to deliver to cells than current CRISPR tools.

A speech-language pathologist by training, Wetherby has spent more than four decades developing tools to help identify and treat autism early; now her work has taken on a more personal sense of urgency.

Children with congenital heart disease have an increased likelihood of autism. Why?

Contrary to conventional wisdom, most people with fragile X syndrome express the FMR1 gene — albeit improperly.

The discovery could help clinicians diagnose children who carry mutations in the gene, called SCN2A, and gauge their responses to potential therapies.

The approach prompts cultured cells to correct the genetic mutation in fragile X syndrome using their own DNA repair system, but it still needs to be tested further.