Preventative treatment of tuberous sclerosis complex with sirolimus: Phase I safety and efficacy results
Jamie K. Capal, David M. Ritter, David Neal Franz, Molly Griffith, Kristn Currans, Bridget Kent, E. Martina Bebin, Hope Northrup, Mary Kay Koenig, Tomoyuki Mizuno, Alexander A. Vinks, Stephanie L. Galandi, Wujuan Zhang, Kenneth D.R. Setchell, Kelly M. Kremer, Carlos M. Prada, Hansel M. Greiner, Katherine Holland-Bouley, Paul S. Horn, Darcy A. Krueger
Ann Child Neurol Soc, 2: 106-119. doi.org/10.1002/cns3.20070
Phase 1 study of preventative sirolimus treatment in tuberous sclerosis complex. Results show that early treatment with sirolimus is safe and the preliminary efficacy is reassuring.
