WHO世界卫生组织：2025年9月镰状细胞疾病儿科药物优化会议报告（英文版）

The paediatric drug optimization (PADO) exercise for sickle cell disease (SCD) aimed to identify short- and long-term therapeutic priorities for children with SCD by reviewing currently approved products and emerging products in the research and development pipeline, including gene therapies. The outcome of this process is intended to inform and support the alignment of funders, procurers, market coordination entities, researchers, innovators, generic manufacturers, product development partne