Targeted Therapies in Neurofibromatosis Type 1.

IF 4.4 3区 医学 Q2 GENETICS & HEREDITY
Aimee A Sato, Dawn Earl, Stephanie E Wallace
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引用次数: 0

Abstract

Neurofibromatosis type 1 (NF1) is a progressive multisystem condition that is characterized by a wide range of clinical manifestations and clinical variability. Individuals with NF1 can be significantly impacted by NF1-related complications, and targeted therapies are emerging. Currently, MEK inhibitors selumetinib and mirdametinib are the only FDA-approved targeted therapies for NF1-related symptomatic or progressive, inoperable plexiform neurofibromas. Several additional MEK inhibitors are being investigated in clinical trials for the treatment of plexiform neurofibromas. Additional therapies are currently under investigation for the treatment of malignant peripheral nerve sheath tumors, low-grade gliomas, skeletal manifestations, cutaneous neurofibromas, and other NF1-related complications.

1型神经纤维瘤病的靶向治疗。
1型神经纤维瘤病(NF1)是一种进行性多系统疾病,具有广泛的临床表现和临床变异性。NF1患者可能会受到NF1相关并发症的显著影响,因此靶向治疗正在出现。目前,MEK抑制剂selumetinib和mirdametinib是fda批准的唯一针对nf1相关症状性或进行性、不能手术的丛状神经纤维瘤的靶向治疗药物。另外几种MEK抑制剂正在临床试验中用于治疗丛状神经纤维瘤。目前正在研究其他治疗恶性周围神经鞘肿瘤、低级别胶质瘤、骨骼表现、皮肤神经纤维瘤和其他nf1相关并发症的方法。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
CiteScore
7.00
自引率
0.00%
发文量
42
审稿时长
>12 weeks
期刊介绍: Seminars in Medical Genetics, Part C of the American Journal of Medical Genetics (AJMG) , serves as both an educational resource and review forum, providing critical, in-depth retrospectives for students, practitioners, and associated professionals working in fields of human and medical genetics. Each issue is guest edited by a researcher in a featured area of genetics, offering a collection of thematic reviews from specialists around the world. Seminars in Medical Genetics publishes four times per year.
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