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Gene therapy for Hurler syndrome

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Several traditional, susceptibleinfectedremoved models have been applied to describe influenza epidemics; however, these models cannot quantitatively assess the efficacy of vaccination. Otani et al.’s study aimed to develop a novel… Click to show full abstract

Several traditional, susceptibleinfectedremoved models have been applied to describe influenza epidemics; however, these models cannot quantitatively assess the efficacy of vaccination. Otani et al.’s study aimed to develop a novel model to virtually reproduce the profiles of seasonal influenza epidemics and to evaluate the efficacy of vaccination based on the pharmacometric modeling technique using realworld surveillance data. Their results showed that influenza type A displayed a higher rate of transmission compared with influenza type B, and the highest rate of influenza transmission and greatest preventive effect of vaccination were observed in children aged 5– 14 years. These were the first quantitative analyses of influenza transmission in the community and of the efficacy of treatment modalities, including vaccination. This modeling approach is applicable to other viral infections. Otani et al. CPT Pharmacometrics Syst. Pharmcol. https://doi.org/10.1002/ psp4.12732. Credit: Shutterstock/VGstockstudio Phase I Trial of BTK Inhibitor Tolebrutinib

Keywords: vaccination; transmission; gene therapy; efficacy; therapy hurler; hurler syndrome

Journal Title: Journal of Paediatrics and Child Health
Year Published: 2021

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