In a phase 2 trial of adults with neurofibromatosis type 1 and inoperable/growing plexiform neurofibromas, treatment with the MEK inhibitor selumetinib resulted in an objective response rate of 63.6% and improvement in other patient outcomes, with additional biopsy-based data providing further information on drug activity.
- Andrea M. Gross
- Geraldine O’Sullivan Coyne
- Brigitte C. Widemann
