Selumetinib in Children with Inoperable Plexiform Neurofibromas

No approved therapies exist for this population. In a phase 2 trial (n=50) of this oral selective MEK inhibitor, 35 patients (70%) with neurofibromatosis type 1 had a confirmed partial response and 28 of these patients had a durable response (lasting ≥1 year).


New England Journal of Medicine