FDA approval of trofinetide may spur further drug development for Rett
The drug, welcomed by patients, might be just the first of many.
Rare or common, inherited or spontaneous, mutations form the core of autism risk.
The drug, welcomed by patients, might be just the first of many.
The most comprehensive study of neurodevelopmental conditions in Kenya and South Africa ever conducted shares preliminary results and lessons.
The proteins are part of a newly discovered complex that mends genetic damage exclusively in neurons.
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.
The OTUD7A gene, which may account for some traits in people missing a segment of chromosome 15, appears to interact with several known autism-linked genes.
FMR1 loss impairs sodium channels, hindering mouse neurons from generating the electrical signals needed to transmit information.
The approach removes methyl tags from the gene and shields it from other silencing factors without changing the gene itself, raising hopes for a new treatment.
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.
Exposing neurons to valproic acid, a well-known environmental risk factor for autism, disrupts their ability to generate different proteins from the same gene.