Real-world outcomes for selumetinib in pediatric patients with neurofibromatosis type 1 and plexiform neurofibromas in Japan: A 1-year interim analysis.

Effectiveness At 1 year, the effectiveness assessments showed a trend toward improvement in the overall evaluation of PN performed by the investigator ( Figure 3A ), decrease in the target PN size on imaging ( Figure 3B ), and patient’s general self-assessment ( Figure 3C ) with selumetinib treatment.

also in 53,322 other papers