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Rapamycin for Autism Phenotype & seizures in Tuberous Sclerosis (RAPS)
Tuberous sclerosis complex (TSC) is associated with autism and seizures. Recent studies in animals using rapamycin have shown cognitive improvement. Human studies have demonstrated a reduction in tumor burden, subjective cognitive improvement, and decreased seizure frequency. We plan to examine the ability of rapamycin to reduce the symptoms of autism, decrease seizure frequency, and improve cognition in patients affected by TSC.
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