April 22, 2024Open Access

Preventative treatment of tuberous sclerosis complex with sirolimus: Phase I safety and efficacy results

JCJamie K. CapalCincinnati Children's Hospital Medical Center DRDavid M. RitterCincinnati Children's Hospital Medical Center DFDavid Neal FranzCincinnati Children's Hospital Medical Center

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Abstract

Tuberous sclerosis complex (TSC) results from overactivity of the mechanistic target of rapamycin (mTOR). Sirolimus and everolimus are mTOR inhibitors that treat most facets of TSC but are understudied in infants. We sought to understand the safety and potential efficacy of preventative sirolimus in infants with TSC.

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Capal et al. (Mon,) studied this question.

synapsesocial.com/papers/68e6e1d5b6db64358765cfc8 https://doi.org/https://doi.org/10.1002/cns3.20070

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