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Review
. 2023 Oct;70(5):937-950.
doi: 10.1016/j.pcl.2023.05.007.

Shedding New Light: Novel Therapies for Common Disorders in Children with Neurofibromatosis Type I

Natasha Pillay-Smiley  1 , Jonathan S Fletcher  2 , Peter de Blank  1 , Nancy Ratner  3
Affiliations
Review

Shedding New Light: Novel Therapies for Common Disorders in Children with Neurofibromatosis Type I

Natasha Pillay-Smiley et al. Pediatr Clin North Am. 2023 Oct.

Abstract

Neurofibromatosis type I (NF1) is a common dominantly inherited disorder, and one of the most common of the RASopathies. Most individuals with NF1 develop plexiform neurofibromas and cutaneous neurofibromas, nerve tumors caused by NF1 loss of function in Schwann cells. Cell culture models and mouse models of NF1 are being used to test drug efficacy in preclinical trials, which led to Food and Drug Administration approval for use of MEK inhibitors to shrink most inoperable plexiform neurofibromas. This article details methods used for testing in preclinical models, and outlines newer models that may identify additional, curative, strategies.

Keywords: Low-grade glioma; MEK inhibitor; Neurofibromatosis type 1; Plexiform neurofibroma.

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