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Method for characterizing the efficacy of an agent targeting a primary cystic fibrosis defect
Method for characterizing the efficacy of an agent targeting a primary cystic fibrosis defect
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机译:表征靶向原发性囊性纤维化缺损的药物功效的方法
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摘要
Generally, the method for characterizing the efficacy of an agent targeting a primary cystic fibrosis defect comprises measuring a change in the status of lung infection in a sample population of subjects administered the agent in comparison to a control sample population of subjects; wherein the subjects in the sample population and the control sample population have the primary cystic fibrosis defect and are uninfected before the agent is administered; wherein a beneficial change in the presence of lung infection in the sample population in comparison to the control sample population is indicative of a treatment effect; and wherein the agent lacks intrinsic antibiotic activity.
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