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doi: 10.1007/s12020-019-01882-4 19 PattisonDJHarrisonRASymmonsDP

15 score (Table 6)

J Pediatr 138:880887

During the initial stages of clinical development, administration of nirogacestat demonstrated activity in patients with DT by inducing a partial response in five and prolonged disease stabilization in two out of seven patients [37, 38]