Charcot-Marie-Tooth 型 1 疾病的治疗进展。
Therapeutic Development in Charcot Marie Tooth Type 1 Disease.
机构信息
InFlectis BioScience SAS, 21 Rue La Noue Bras de Fer, 44200 Nantes, France.
Centre de recherche en CardioVasculaire et Nutrition, Aix-Marseille Université, INRA 1260-INSERM 1263, 13005 Marseille, France.
出版信息
Int J Mol Sci. 2021 Jun 23;22(13):6755. doi: 10.3390/ijms22136755.
Charcot-Marie-Tooth disease (CMT) is the most frequent hereditary peripheral neuropathies. It is subdivided in two main groups, demyelinating (CMT1) and axonal (CMT2). CMT1 forms are the most frequent. The goal of this review is to present published data on 1-cellular and animal models having opened new potential therapeutic approaches. 2-exploration of these tracks, including clinical trials. The first conclusion is the great increase of publications on CMT1 subtypes since 2000. We discussed two points that should be considered in the therapeutic development toward a regulatory-approved therapy to be proposed to patients. The first point concerns long term safety if treatments will be a long-term process. The second point relates to the evaluation of treatment efficiency. Degradation of CMT clinical phenotype is not linear and progressive.
Charcot-Marie-Tooth 病（CMT）是最常见的遗传性周围神经病。它分为两个主要类型，脱髓鞘（CMT1）和轴索（CMT2）。CMT1 型最为常见。本文的目的是介绍在单细胞和动物模型方面的发表数据，这些模型为潜在的治疗方法开辟了新的途径。探讨这些途径，包括临床试验。第一个结论是，自 2000 年以来，CMT1 亚型的出版物数量大幅增加。我们讨论了在治疗开发中应考虑的两个问题，以便向患者提出经监管机构批准的治疗方法。第一个问题涉及到如果治疗是一个长期过程，长期安全性。第二个问题涉及到治疗效果的评估。CMT 临床表型的退化不是线性和进行性的。
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