Results from expanded access programs: a review of academic literature 167 7♦ the efficacy of nirogacestat, and additional studies should be undertaken to establish the feasibility of this approach in other patients’. The authors primarily note the ‘small number of patients’, ‘lack of controls’ and therefore call for ‘further investigation is needed to understand its efficacy and safety in pediatric patients’. Conclusion Based on the observed responses, the authors conclude that ‘nirogacestat is a promising option for treating pediatric patients with desmoid tumors’.
RkJQdWJsaXNoZXIy MTk4NDMw