Safety and efficacy of selumetinib in pediatric and adult patients with neurofibromatosis type 1 and plexiform neurofibroma.
Hyery KimHee Mang YoonEun Key KimYoung Shin RaHyo-Won KimMi-Sun YumMin-Jee KimJae Suk BaekYu Sub SungSang Min LeeHyeong-Seok LimByung Joo LeeHyun Taek LimDohyung KimJihee YoonHyunwoo BaeSoojin HwangYun-Ha ChoiKyung Ah KimIn Hee ChoiSeung Won LeeSu-Jung ParkBeom-Hee LeePublished in: Neuro-oncology (2024)
Selumetinib decrease PN volume in the majority of pediatric and adult NF1 patients while also showing efficacy in non-malignant diverse NF1 manifestations.