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Journal Articles International Journal of Molecular Sciences Year : 2021

Therapeutic Development in Charcot Marie Tooth Type 1 Disease


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.
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hal-03769789 , version 1 (05-09-2022)


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Pierre Miniou, Michel Fontes. Therapeutic Development in Charcot Marie Tooth Type 1 Disease. International Journal of Molecular Sciences, 2021, 22 (13), pp.6755. ⟨10.3390/ijms22136755⟩. ⟨hal-03769789⟩
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