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A clinical trial testing an old drug on children with a rare genetic condition called tuberous sclerosis complex is drawing attention because the disease often leads to autism.
Up to half the people with tuberous sclerosis develop autism, and the hope is that any treatment that works well against tuberous sclerosis might also benefit others on the autism spectrum.
At the very least, doctors hope that by researching the rarer condition—which strikes about 1 in 6,000 newborns and spurs the growth of benign tumors—they will gain insights into autism. The government estimates that autism affects 1 in 50 children.
In the Boston Children’s Hospital study, 50 children with tuberous sclerosis are taking a drug similar to rapamycin—used for decades to prevent rejection of transplanted organs—or a placebo.