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Widespread suppression of huntingtin with convection-enhanced delivery of siRNA.
Stiles DK, Zhang Z, Ge P, Nelson B, Grondin R, Ai Y, Hardy P, Nelson PT, Guzaev AP, Butt MT, Charisse K, Kosovrasti V, Tchangov L, Meys M, Maier M, Nechev L, Manoharan M, Kaemmerer WF, Gwost D, Stewart GR, Gash DM, Sah DW. Stiles DK, et al. Exp Neurol. 2012 Jan;233(1):463-71. doi: 10.1016/j.expneurol.2011.11.020. Epub 2011 Nov 19. Exp Neurol. 2012. PMID: 22119622
A phase 3 study of tezacaftor in combination with ivacaftor in children aged 6 through 11 years with cystic fibrosis.
Walker S, Flume P, McNamara J, Solomon M, Chilvers M, Chmiel J, Harris RS, Haseltine E, Stiles D, Li C, Ahluwalia N, Zhou H, Owen CA, Sawicki G; VX15-661-113 Investigator Group. Walker S, et al. J Cyst Fibros. 2019 Sep;18(5):708-713. doi: 10.1016/j.jcf.2019.06.009. Epub 2019 Jun 26. J Cyst Fibros. 2019. PMID: 31253540 Free article. Clinical Trial.
Safety, pharmacokinetics, and pharmacodynamics of lumacaftor and ivacaftor combination therapy in children aged 2-5 years with cystic fibrosis homozygous for F508del-CFTR: an open-label phase 3 study.
McNamara JJ, McColley SA, Marigowda G, Liu F, Tian S, Owen CA, Stiles D, Li C, Waltz D, Wang LT, Sawicki GS. McNamara JJ, et al. Lancet Respir Med. 2019 Apr;7(4):325-335. doi: 10.1016/S2213-2600(18)30460-0. Epub 2019 Jan 24. Lancet Respir Med. 2019. PMID: 30686767 Clinical Trial.