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Day One Biopharmaceuticals said Sunday that a targeted therapy shrank tumors by at least 50% in a majority of children with the most common form of pediatric brain cancer, setting up a potential approval in a rare disease long overlooked by drugmakers. 

The new data expand on earlier results released last year from 22 patients. Of the 69 pediatric low-grade glioma patients now evaluated, 44, or 64%, saw their tumors dwindle to less than half their original size, including three patients whose tumors disappeared. 


The patients’ responses lasted a median of 8.4 months, although that number may rise as the trial continues. As of Sept. 28, when the data were collected, 77% of patients were still taking the drug, known as tovorafenib, the company said in a press release.

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