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    • Safety, pharmacokinetics, and preliminary assessment of efficacy of mecasermin (recombinant human IGF-1) for the treatment of Rett syndrome 

      Khwaja, O. S.; Ho, E.; Barnes, K. V.; O, H. M.; Pereira, Luis Marcelo; Finkelstein, Y.; Nelson, Charles A.; Vogel-Farley, V.; DeGregorio, G.; Holm, Ingrid Adele; Khatwa, Umakanth A.; Kapur, Kush; Alexander, Mark Edward; Finnegan, D. M.; Cantwell, N. G.; Walco, A. C.; Rappaport, Leonard Allan; Gregas, M.; Fichorova, Raina Nakova; Shannon, M. W.; Sur, M.; Kaufmann, Walter E (Proceedings of the National Academy of Sciences, 2014)
      Rett syndrome (RTT) is a severe X-linked neurodevelopmental disorder mainly affecting females and is associated with mutations in MECP2, the gene encoding methyl CpG-binding protein 2. Mouse models suggest that recombinant ...