国会预算办公室：2025年增加镰状细胞病基因治疗的使用将如何影响联邦预算报告（英文版）

Gene therapies replace or modify disease-causing genes in human cells. Those therapies, which are still relatively new, are used to prevent, cure, or treat the symptoms of a particular disease. In this report, the Congressional Budget Office discusses how it would estimate the budgetary effects of policies that sought to increase the use of gene therapy treatment for sickle cell disease (SCD). CBO focuses on SCD because two new gene therapies were recently approved for its treatment, and the fir