bidridistrogene xeboparvovec 治疗 2E/R4 型肢带型肌营养不良症的基因治疗:1/2 期试验结果。 21.The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial. II 型 RAF 抑制剂托沃拉非尼治疗复发/难治性儿童低级别胶质瘤:2 期 FIREFLY-1 试验。
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Functional precision medicine for pediatric cancers A small, prospective clinical study shows that ex vivo drug screening of pediatric cancer samples can identify effective therapeutic options. If validated, these findings could herald a new approach to precision medicine in this setting. M. Emmy M....