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Preventative treatment of tuberous sclerosis complex with sirolimus: Phase I safety and efficacy results.
Capal JK, Ritter DM, Franz DN, Griffith M, Currans K, Kent B, Bebin EM, Northrup H, Koenig MK, Mizuno T, Vinks AA, Galandi SL, Zhang W, Setchell KDR, Kremer KM, Prada CM, Greiner HM, Holland-Bouley K, Horn PS, Krueger DA. Capal JK, et al. Ann Child Neurol Soc. 2024 Jun;2(2):106-119. doi: 10.1002/cns3.20070. Epub 2024 Apr 22. Ann Child Neurol Soc. 2024. PMID: 39726432 Free PMC article.