Article
The Current State of Charcot-Marie-Tooth Disease Treatment.
Genes - 1 Jul 2023
Okamoto Yuji, Takashima Hiroshi
Abstract excerpt
Charcot-Marie-Tooth disease (CMT) and associated neuropathies are the most predominant genetically transmitted neuromuscular conditions; however, effective pharmacological treatments have not established. The extensive genetic heterogeneity of CMT, which impacts the peripheral nerves and causes lifelong disability, presents a significant barrier to the development of comprehensive treatments. An estimated 100...
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