The approach improves the function of SYNGAP1-deficient neurons in vitro, but whether it will work in people remains unclear.
Spectrum: Autism Research News
Efforts to ease the symptoms of autism are beginning to ramp up, with promising candidates in various stages of testing.
The drug, welcomed by patients, might be just the first of many.
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.