The DOI matches its Crossref record; 104 of the 104 references it cites were found in a registry or an index.
Drug Repurposing for Rare Diseases
Open accessAbstract
Currently, there are about 7000 identified rare diseases, together affecting 10% of the population. However, fewer than 6% of all rare diseases have an approved treatment option, highlighting their tremendous unmet needs in drug development. The process of repurposing drugs for new indications, compared with the development of novel orphan drugs, is a time-saving and cost-efficient method resulting in higher success rates, which can therefore drastically reduce the risk of drug development for rare diseases. Although drug repurposing is not novel, new strategies have been developed in recent years to do it in a systematic and rational way. Here, we review applied methodologies, recent accomplished progress, and the challenges associated in drug repurposing for rare diseases.
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Cites
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Cited by
Advance in peptide-based drug development: delivery platforms, therapeutics and vaccines
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AlphaFold, Artificial Intelligence (AI), and Allostery
Nussinov, R., Zhang, M., Liu, Y., & Jang, H. (2022) · The Journal of Physical Chemistry B · doi:10.1021/acs.jpcb.2c04346 · Cited by 190
Adachi, T., El-Hattab, A. W., Jain, R., Nogales Crespo, K. A., Quirland Lazo, C. I., Scarpa, M., Summar, M., & Wattanasirichaigoon, D. (2023) · International Journal of Environmental Research and Public Health · doi:10.3390/ijerph20064732 · Cited by 168
“This is a timesaving, cost-efficient method that can accelerate the development of RDs treatments [59].”
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Defelippe, V. M., J.M.W. van Thiel, G., Otte, W. M., Schutgens, R. E. G., Stunnenberg, B., Cross, H. J., O'Callaghan, F., De Giorgis, V., Jansen, F. E., Perucca, E., et al. (2023) · Drug Discovery Today · doi:10.1016/j.drudis.2023.103688 · Cited by 30
Metadata
- Authors
- Roessler, H. I., Knoers, N. V. A. M., van Haelst, M. M., & van Haaften, G.
- Venue
- Trends in Pharmacological Sciences
- Publisher
- Elsevier BV
- Published
- 2021-04
- Volume
- 42
- Issue
- 4
- Pages
- 255-267
- ISSN
- 0165-6147 (ISSN-L)
- OpenAlex
- W3127311830
- Cited by
- 269
- References
- 104
- FWCI
- 4.37
- Type
- journal article
- Language
- en
- Open access
- hybrid
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- Roessler, H. I., Knoers, N. V. A. M., van Haelst, M. M., & van Haaften, G. (2021). Drug Repurposing for Rare Diseases. Trends in Pharmacological Sciences. https://doi.org/10.1016/j.tips.2021.01.003
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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.
