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 accessAbstract
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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Citations
Cites
Report of the committee on methods of clinical examination in electroencephalography
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Reversal of Neurological Defects in a Mouse Model of Rett Syndrome
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A Wearable Sensor for Unobtrusive, Long-Term Assessment of Electrodermal Activity
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Partial reversal of Rett Syndrome-like symptoms in MeCP2 mutant mice
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Self-citationClinical manifestations and stages of rett syndrome
Hagberg, B. (2002) · Mental Retardation and Developmental Disabilities Research Reviews · doi:10.1002/mrdd.10020 · Cited by 416
Berry-Kravis, E. M., Hessl, D., Rathmell, B., Zarevics, P., Cherubini, M., Walton-Bowen, K., Mu, Y., Nguyen, D. V., Gonzalez-Heydrich, J., Wang, P. P., et al. (2012) · Science Translational Medicine · doi:10.1126/scitranslmed.3004214 · Cited by 328
A Comparison of the Electroencephalogram between Institutionalized and Community Children in Romania
Marshall, P. J., Fox, N. A., & Group, B. C. (2004) · Journal of Cognitive Neuroscience · doi:10.1162/0898929042304723 · Cited by 268
Henderson, C., Wijetunge, L., Kinoshita, M. N., Shumway, M., Hammond, R. S., Postma, F. R., Brynczka, C., Rush, R., Thomas, A., Paylor, R., et al. (2012) · Science Translational Medicine · doi:10.1126/scitranslmed.3004218 · Cited by 249
Functional recovery with recombinant human IGF1 treatment in a mouse model of Rett syndrome
Castro, J., Garcia, R. I., Kwok, S., Banerjee, A., Petravicz, J., Woodson, J., Mellios, N., Tropea, D., & Sur, M. (2014) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1311685111 · Cited by 242
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Cited by
Genes, circuits, and precision therapies for autism and related neurodevelopmental disorders
Sahin, M., & Sur, M. (2015) · Science · doi:10.1126/science.aab3897 · Cited by 295
Self-citationMellios, N., Feldman, D. A., Sheridan, S. D., Ip, J. P. K., Kwok, S., Amoah, S. K., Rosen, B., Rodriguez, B. A., Crawford, B., Swaminathan, R., et al. (2018) · Molecular Psychiatry · doi:10.1038/mp.2017.86 · Cited by 292
Self-citationModeling non-syndromic autism and the impact of TRPC6 disruption in human neurons
Griesi-Oliveira, K., Acab, A., Gupta, A. R., Sunaga, D. Y., Chailangkarn, T., Nicol, X., Nunez, Y., Walker, M. F., Murdoch, J. D., Sanders, S. J., et al. (2015) · Molecular Psychiatry · doi:10.1038/mp.2014.141 · Cited by 236
Functional recovery with recombinant human IGF1 treatment in a mouse model of Rett syndrome
Castro, J., Garcia, R. I., Kwok, S., Banerjee, A., Petravicz, J., Woodson, J., Mellios, N., Tropea, D., & Sur, M. (2014) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1311685111 · Cited by 242
Self-citationMECP2 disorders: from the clinic to mice and back
Lombardi, L. M., Baker, S. A., & Zoghbi, H. Y. (2015) · Journal of Clinical Investigation · doi:10.1172/jci78167 · Cited by 221
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
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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.