DeFi trial: Smart design and impressive results for the benefit of patients with rare cancers

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DeFi trial: Smart design and impressive results for the benefit of patients with rare cancers

Significant improvement in progression free survival and response rate combined with reduced symptoms and better quality of life are reported as outcomes of a new treatment approach for patients with desmoid tumors, which are benign, but locally aggressive and invasive soft tissue tumors. By targeting the Notch pathway with the novel gamma secretase inhibitor nirogacestat, researchers from the DeFi trial have obtained positive results for the first time with this approach. The results are reported at the ESMO Congress 2022.

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