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Trametinib therapy for children with neurofibromatosis type 1 and life-threatening plexiform neurofibroma or treatment-refractory low-grade glioma.

Rebecca RonsleyCeline D HounjetSylvia ChengShahrad Rod RassekhWalter J DuncanChristopher DunhamJane GardinerArvindera GhagJeffrey P LudemannDavid WensleyWingfield RehmusMichael A SargentJuliette Hukin
Published in: Cancer medicine (2021)
Trametinib is an effective therapy for advanced PNF and refractory PLGG in patients with NF-1 and is well tolerated in children. Further data and clinical trials are required to assess tolerance, efficacy and durability of response, and length of treatment required in such patients.
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