Several targeted trials on drugs that treat fragile X syndrome are under way. But accurate endpoints to measure the drugs’ effectiveness are crucial, argues developmental and behavioral pediatrician Randi Hagerman.

A new opinion piece argues that the risks of early screening for autism outweigh the benefits. But the article does a poor job of articulating what these risks might be, instead focusing only on the fact that there are few good options available to children after their diagnosis.

Researchers have identified hundreds of previously unknown connections between proteins involved in autism spectrum disorders, according to a report published last week in Science Translational Medicine.

A drug called arbaclofen has matriculated to a phase III clinical trial, the last and most difficult step on the long road to regulatory approval.

Social media is powerful, and scientists can and should harness it to help answer difficult questions. But as with any easy solution, there is a corresponding cautionary tale: Social media can just as easily misguide as be meaningful.

Only a small fraction of autism therapies are supported by robust scientific evidence, according to three reviews published in the May issue of Pediatrics.

Practical and ethical challenges in translational research could be better overcome if clinical researchers were to add genetic counselors to their teams, a new report argues.

Hallucinogens like LSD and MDMA may help people with autism become more sociable, but negative side effects argue against their use.

The National Institutes of Health is adding a new $1 billion institute for early-stage drug development. But the agency’s plan to fund it by closing the National Center for Research Resources is causing some consternation.

As more drugs enter clinical trials for fragile X syndrome and soon, hopefully, for autism, the placebo effect will become an important consideration.