RNA therapy restores gene function in monkeys modeling Angelman syndrome
The result raises hopes for an ongoing clinical trial in people — and offers fresh insight into the biology of imprinting and the UBE3A antisense transcript.
Efforts to ease the symptoms of autism are beginning to ramp up, with promising candidates in various stages of testing.
The result raises hopes for an ongoing clinical trial in people — and offers fresh insight into the biology of imprinting and the UBE3A antisense transcript.
The changes may help explain the link between maternal infection and autism, though more research is needed.
Some conditions are too rare for conventional drug trials, leading some scientists to test bespoke treatments in single participants. Gleeson discusses the merits — and limitations — of these tiny trials.
This month’s issue of the Going on Trial newsletter examines personalized therapies for rare conditions, Acadia’s new drug for Rett syndrome and developments in a cord-blood program, among other autism-related drug trial news.
Studies of behavioral treatments for autism are complex and can easily be misunderstood. Here we provide some guidance.
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.