Data on Neurofibromatosis Type 1 Published by a Researcher at University of Ulsan College of Medicine (Safety and efficacy of selumetinib in pediatric and adult patients with neurofibromatosis type 1 and plexiform neurofibroma).

A recent study conducted by researchers at the University of Ulsan College of Medicine in Seoul, South Korea, has found that the MEK inhibitor selumetinib can reduce plexiform neurofibroma (PN) in pediatric patients with neurofibromatosis type 1 (NF1). The study enrolled 90 pediatric and adult NF1 patients with inoperable, symptomatic, or potentially morbid PN and administered selumetinib for 2 years. The results showed that selumetinib led to a reduction in PN volume in the majority of patients, as well as improvements in neurocognitive function, growth parameters, cafe-au-lait spots, and quality of life. The study concluded that selumetinib is effective in treating diverse manifestations of NF1. [Extracted from the article]