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Tuberous sclerosis complex

Kellen Winden1, E Martina Bebin2, Shafali Jeste3

  • 1Department of Neurology, Rosamund Stone Zander and Hansjoerg Wyss Translational Neuroscience Center, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.

Nature Reviews. Disease Primers
|March 13, 2026
PubMed
Summary

Tuberous sclerosis complex (TSC) is a rare genetic disorder. Rapalogues treat TSC manifestations, but TAND and refractory epilepsy remain challenging, highlighting an unmet therapeutic need.

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