Vincent et al. report the effect of MEK inhibitor trametinib in two children with syndromic KRAS-related multiple non-ossifying fibromas (NOFs). This treatment in vitro reduces KRAS pathway hyperactivation and in vivo induces reossification of NOFs, thus representing a promising targeted therapy for severe KRAS-related bone fragility.
- Marie Vincent
- Soizic Tiriau
- Massimiliano Rossi
