Bioscience Evidence 2024, Vol.14, No.1, 16-23 http://bioscipublisher.com/index.php/be 22 Chakravarty K., Antontsev V., Bundey Y., and Varshney J., 2021, Driving success in personalized medicine through AI-enabled computational modeling, Drug Discovery Today, 26(6): 1459-1465. https://doi.org/10.1016/j.drudis.2021.02.007 Chen X., Liu L.H., and Wang T., 2022, Regulatory evaluation of drugs for the treatment of rare disease by taking Risdiplam as an example, Zhongguo Lingchuang Yaolixue Zazhi (Chin J Clin Pharmacol), 38(8): 883-888. Goetz L.H., and Schork N.J., 2018, Personalized medicine: motivation, challenges, and progress, Fertility and sterility, 109(6): 952-963. https://doi.org/10.1016/j.fertnstert.2018.05.006 Groft S.C., Posada M., and Taruscio D., 2021, Progress, challenges and global approaches to rare diseases, Acta Paediatrica, 110(10): 2711-2716. https://doi.org/10.1111/apa.15974 Kempf L., Goldsmith J., and Temple R., 2018, Challenges of developing and conducting clinical trials in rare disorders, American Journal of Medical Genetics Part A, 176(4): 773-783. https://doi.org/10.1002/ajmg.a.38413 Kubota K., Funabashi M., and Ogura Y., 2019, Target deconvolution from phenotype-based drug discovery by using chemical proteomics approaches, Biochimica et Biophysica Acta (BBA) - Proteins and Proteomics, 1867(1): 22-27. https://doi.org/10.1016/j.bbapap.2018.08.002 Li Z.Y., Huang Y.Q., Zhou Y.H., Mao Y.Y., and Gao L.B., 2018, Hot topics in drug research and development in 2017: Biological drug rapid growth, breakthrough therapy certification, Keji Daobao (Science & Technology Review), 36(1): 116-125. Lim H., Poleksic A., Yao Y., Tong H., He D., Zhuang L., Meng P., and Xie L., 2016, Large-Scale Off-Target Identification using fast and accurate dual regularized one-class collaborative filtering and its application to drug repurposing, PLoS Computational Biology, 12(10): e1005135. https://doi.org/10.1371/journal.pcbi.1005135 Liu J., Barrett J.S., Leonardi E.T., Lee L., Roychoudhury S., Chen Y., and Trifillis P., 2022, Natural history and real-world data in rare diseases: applications, limitations, and future perspectives, The Journal of Clinical Pharmacology, 62: 38-55. https://doi.org/10.1002/jcph.2134 Miller K.L., Fermaglich L.J., and Maynard J., 2021, Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases,. Orphanet journal of rare diseases, 16(1): 1-10. https://doi.org/10.1186/s13023-021-01901-6 Nestler-Parr S., Korchagina D., Toumi M., Pashos C.L., Blanchette C., Molsen E., Morel T., Simoens S., Kaló Z., Gatermann R., and Redekop W., 2018, Challenges in research and health technology assessment of rare disease technologies: report of the ISPOR rare disease special interest group, Value in Health, 21(5): 493-500. https://doi.org/10.1016/j.jval.2018.03.004 Polizzi A., Balsamo A., Bal M.O., and Taruscio D., 2014, Rare diseases research and practice, Understanding Differences and Disorders of Sex Development (DSD), 27: 234-256. https://doi.org/10.1159/000363670 Qiu T., Wang Y., Dabbous M., Hanna E., Han R., Liang S., and Toumi M., 2020, Current state of developing advanced therapies for rare diseases in the European Union, Expert Opinion on Orphan Drugs, 8(10): 417-429. https://doi.org/10.1080/21678707.2020.1835640 Smith S.C., 2022, A zebra's trust: How rare disease communities' participation in data trusts' governance builds trust and drives research, Vand. J. Transnat'l L., 55: 191. Sun W., Zheng W., and Simeonov A., 2017, Drug discovery and development for rare genetic disorders, American Journal of Medical Genetics Part A, 173: 2307-2322. https://doi.org/10.1002/ajmg.a.38326 Tang L., Song Y., Yang Z., 2022, Consideration on the research and development of new drugs for rare tumors, Zhongguo Feiai Zazhi (Chin J Lung Cancer), 25(7): 443-447. Tang Y., Zhang X.J., Liu X., Mei D., Fu Q., Li J.T., Zhang Q., Zhang P.X., Zuo W., and Zhang B., 2020, The enlightenment of foreign drug security system for rare diseases to China, zhongguo Yaoxue Zazhi (Chinese Pharmaceutical Journal), 55(11): 955-960. Visibelli A., Roncaglia B., Spiga O., and Santucci A., 2023, The impact of artificial intelligence in the odyssey of rare diseases, Biomedicines, 11(3): 887. https://doi.org/10.3390/biomedicines11030887 Viviani L., Harrison M.J., Zolin A., Haworth C.S., and Floto A., 2016, Epidemiology of nontuberculous mycobacteria (NTM) amongst individuals with cystic fibrosis (CF), J. Cyst. Fibros., 15(5): 619-623. https://doi.org/10.1016/j.jcf.2016.03.002 Walters W., and Barzilay R., 2021, Critical assessment of AI in drug discovery, Expert Opinion on Drug Discovery, 16(9): 937-947. https://doi.org/10.1080/17460441.2021.1915982 Weng H.B., Chen H.X., and Wang M.W., 2018, Innovation in neglected tropical disease drug discovery and development, Infectious diseases of poverty, 7(3): 1-9. https://doi.org/10.1186/s40249-018-0444-1 Yang Z.X., Li Y., and Ding W.X., 2023, Analysis on research and development police and status of drug for rare diseases in China, Yaowu Pingjia Yanjiu (Drug
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