Abstract
Background: Tuberous sclerosis complex (TSC) is a multisystem disease with age-dependent development of hamartomas in multiple organic systems. Complications in neonates and infants occur by cardiac rhabdomyomas causing hemodynamic instability, arrhythmias or other complications and by subependymal giant cell astrocytomas (SEGA) causing obstructive hydrocephalus. A targeted therapy with everolimus, a selective mTOR inhibitor is licensed in Europe and USA for the treatment of SEGA and angiomyolipomas at the age of 3 years.
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