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

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

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 human insulin-like growth factor 1 (IGF-1) (rhIGF1) (mecasermin) may improve...

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Main Authors: Khwaja, Omar S. (Author), Ho, Eugenia (Author), Barnes, Katherine V. (Author), O'Leary, Heather M. (Author), Pereira, Luis M. (Author), Finkelstein, Yaron (Author), Nelson III, Charles A. (Author), Vogel-Farley, Vanessa (Author), DeGregorio, Geneva (Author), Holm, Ingrid A. (Author), Khatwa, Umakanth (Author), Kapur, Kush (Author), Alexander, Mark E. (Author), Finnegan, Deidre M. (Author), Cantwell, Nicole G. (Author), Walco, Alexandra C. (Author), Rappaport, Leonard (Author), Gregas, Matt (Author), Fichorova, Raina N. (Author), Shannon, Michael W. (Author), Sur, Mriganka (Contributor), Kaufmann, Walter E. (Author)
Other Authors: Massachusetts Institute of Technology. Department of Brain and Cognitive Sciences (Contributor)
Format: Article
Language:English
Published: National Academy of Sciences (U.S.), 2014-11-06T18:21:18Z.
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