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Patients commonly present with progressive weakness and atrophy of the distal muscles, accompanied by hyperalgesia, decreased or absent tendon reflexes, and foot deformities. Current clinical management relies on multidisciplinary supportive care. Recent preclinical studies targeting potential therapeutic strategies for CMT1A have focused on correcting the gene-dose imbalance of\n                    <jats:italic>PMP22<\/jats:italic>\n                    . Notably, PXT3003 has shown phase III clinical efficacy in relieving symptoms and reducing neuropathy, and is expected to be the earliest CMT1A-targeted drug on the market. Gene editing approaches have also shown therapeutic promise in animal models, but off-target effects remain a concern. In addition, the rapid development of induced pluripotent stem cell (iPSC) technology has paved the way for stem cell therapies, which may be a promising therapeutic approach. This article reviews the existing literature on therapeutic strategies for CMT1A and aims to provide a valuable reference for the clinical treatment of CMT1A.\n                  <\/jats:p>","DOI":"10.1007\/s13760-025-02881-1","type":"journal-article","created":{"date-parts":[[2025,8,27]],"date-time":"2025-08-27T14:25:59Z","timestamp":1756304759000},"page":"1525-1533","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["The current status of Charcot-Marie-Tooth disease type 1\u00a0A treatment"],"prefix":"10.1007","volume":"125","author":[{"given":"Hongdan","family":"Qi","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xin","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bing","family":"Wu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jing","family":"Chen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gang","family":"Zhang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,8,27]]},"reference":[{"key":"2881_CR1","doi-asserted-by":"publisher","DOI":"10.1016\/j.neulet.2020.135357","volume":"742","author":"M Stavrou","year":"2021","unstructured":"Stavrou M, Sargiannidou I, Christofi T, Kleopa KA (2021) Genetic mechanisms of peripheral nerve disease. 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