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...
Topics
- Charcot-Marie-Tooth Disease
- Humans
- Mutation
- Intracellular Space
- Calcium
- Gene Silencing
- Genetic Therapy
- Animals
