Cara Westmark has spent the past year building the case that a drug designed for fragile X syndrome might help coronavirus patients, too.
A gene therapy for Angelman syndrome stands at the forefront of efforts to treat autism-linked conditions that stem from single genes.
Drug after drug has failed in clinical trials for fragile X syndrome. A new study provides an explanation — a decrease in the drugs’ effectiveness over time — and points to a new strategy that could circumvent this problem.
Many drugs for fragile X syndrome have failed in large clinical trials, but candidates that target new aspects of the condition may fare better.
Autism and intelligence share genetic variants, researchers grow Neanderthal mini-brains and see overlap with autism, and maternal diabetes is an autism risk factor.
Many people with fragile X syndrome show average rates of protein production, challenging a long-held assumption about the condition.
The drug mavoglurant has no effect on a brain circuit involved in social behavior in a mouse model of fragile X syndrome. That may explain its poor performance in people with the condition.
A drug that selectively blocks one form of an enzyme called GSK-3 prevents seizures and improves cognitive deficits in a mouse model of fragile X syndrome.