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September 20, 2025American Journal of Medical Genetics Part C Seminars in Medical Genetics

Targeted Therapies in Neurofibromatosis Type 1

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Authors

ASAimee SatoSeattle Children's HospitalDEDawn EarlCedars-Sinai Medical CenterSWS WallaceLeiden University

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Overview

Emerging targeted therapies show promise for plexiform neurofibromas and other complications of NF1.

Key Points

  • Targeted therapies have shown potential in treating complications of neurofibromatosis type 1 (NF1), especially plexiform neurofibromas.
  • The FDA has approved MEK inhibitors selumetinib and mirdametinib for inoperable plexiform neurofibromas.
  • Clinical trials are currently exploring additional MEK inhibitors for NF1-related complications, including low-grade gliomas.
  • Research is ongoing to evaluate therapies for various NF1-related conditions, highlighting the need for continued innovation.

Cite This Study

Sato et al. (2025) studied this question.

synapsesocial.com/papers/68d46fcd31b076d99fa69ea6https://doi.org/10.1002/ajmg.c.32151
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1MEK Inhibitors for Neurofibromatosis Type 1-Associated Central and Peripheral Nervous System Tumors2024
  2. 2Neurofibromatosis Type 1: Genetic Mechanisms and Advances in Therapeutic Innovation2025
  3. 3Neurofibromatosis Type 1: Genetic Mechanisms and Advances in Therapeutic Innovation2025 · 7 citations
  4. 4Targeting the extracellular matrix for NF1-associated neurofibroma treatment2024
  5. 5Neurofibromatosis Type 1 and MEK Inhibition: A Comprehensive Review with Focus on Selumetinib Therapy2025 · 5 citations