In a recent, small, randomized trial of 23 children (ages 3 months to 12 years) with TSC, treatment with sirolimus decreased overall seizure frequency 41% over standard of care, but this change failed to reach statistical significance ( p = .11) [ 55 ].
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mTOR inhibitors in the pharmacologic management of tuberous sclerosis complex and their potential role in other rare neurodevelopmental disorders.
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