SAVANNAH, Georgia — There’s no medical treatment for Charcot-Marie-Tooth (CMT) disease, a debilitating neurologic disorder that’s both progressive and incurable. But now, nerve specialists learned, new potential treatments are moving closer to clinical trials. Genetic-based therapies are currently in preclinical research phases, and an experimental small-molecule drug has reached phase 3 in humans, neurologists told...
Tag: <span>Charcot-Marie-Tooth disease</span>
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Biochemists reveal the cause of Charcot-Marie-Tooth disease—too much of a good thing
Vanderbilt researchers—including Charles Sanders, associate dean for research and professor of biochemistry, and graduate student Justin Marinko—have illuminated the cause of Charcot-Marie-Tooth disease, putting them on the road to developing therapeutic approaches for the disease that affects one in 2,500 people. The discovery was published in the article “Direct Relationship Between Increased Expression and Mistrafficking of the Charcot-Marie-Tooth-Associated Protein PMP22” published in...