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Cited by 209

The DOI matches its Crossref record; 12 of the 12 references it cites were found in a registry or an index.

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

Open access
Khwaja, Omar S., Ho, Eugenia, Barnes, Katherine V., O’Leary, Heather M., Pereira, Luis M., Finkelstein, Yaron, Nelson, Charles A., Vogel-Farley, Vanessa, DeGregorio, Geneva, Holm, Ingrid A., Khatwa, Umakanth, Kapur, Kush, Alexander, Mark E., Finnegan, Deirdre M., Cantwell, Nicole G., Walco, Alexandra C., Rappaport, Leonard, Gregas, Matt, Fichorova, Raina N., Shannon, Michael W., Sur, Mriganka, Kaufmann, Walter E.·2014·Proceedings of the National Academy of Sciences

Abstract

Significance: This paper provides unique insights into mechanism-based therapeutics for Rett syndrome (RTT), a devastating neurodevelopmental disorder. This clinical trial was based on pioneer preclinical work from the laboratory of M.S. Outcome measures include clinical instruments, standardized behavioral measures, and biomarkers, the latter being not only objective but also applicable to experimental studies. We believe this work will a have major impact on the understanding and treatment of RTT, as well as other neurodevelopmental disorders.

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Integrity signals

Registry

Crossref · 10.1073/pnas.1311141111

Cites

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Citations

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Metadata

Authors
Khwaja, O. S., Ho, E., Barnes, K. V., O’Leary, H. M., Pereira, L. M., Finkelstein, Y., Nelson, C. A., Vogel-Farley, V., DeGregorio, G., Holm, I. A., Khatwa, U., Kapur, K., Alexander, M. E., Finnegan, D. M., Cantwell, N. G., Walco, A. C., Rappaport, L., Gregas, M., Fichorova, R. N., Shannon, M. W., Sur, M., & Kaufmann, W. E.
Venue
Proceedings of the National Academy of Sciences
Published
2014-03-12
Volume
111
Issue
12
Pages
4596-4601
ISSN
  • 0027-8424 (ISSN-L)
  • 1091-6490
OpenAlex
W2109555365
Cited by
209
References
12
FWCI
14.27
Type
journal article
Language
en
Open access
bronze

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CoChat. (2026). Citation check: Safety, pharmacokinetics, and preliminary assessment of efficacy of mecasermin (recombinant human IGF-1) for the treatment of Rett syndrome. https://app.cochat.ai/papers/10.1073/pnas.1311141111
The paper
Khwaja, O. S., Ho, E., Barnes, K. V., O’Leary, H. M., Pereira, L. M., Finkelstein, Y., Nelson, C. A., Vogel-Farley, V., DeGregorio, G., Holm, I. A., Khatwa, U., Kapur, K., Alexander, M. E., Finnegan, D. M., Cantwell, N. G., Walco, A. C., Rappaport, L., Gregas, M., Fichorova, R. N., . . . Kaufmann, W. E. (2014). Safety, pharmacokinetics, and preliminary assessment of efficacy of mecasermin (recombinant human IGF-1) for the treatment of Rett syndrome. Proceedings of the National Academy of Sciences. https://doi.org/10.1073/pnas.1311141111

Data sources

Bibliographic data: Semantic Scholar, OpenAlex, Crossref.

Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).

Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.