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Clinical Trials: A Primer! In addition to our regular features, read about “A Clinical Trial for Caroline,” fast tracking and more.

Caroline’s journey with a rare and serious form of CMT began when she was just 9 months old. Today, a cure for Caroline may open the door to treatments and a cure for CMT!

Mutations in the gene coding for the gap junction beta-1 protein (GJB1), also known as connexin 32 (Cx32), are associated with CMT1X. Dr. Kleopa will test whether delivery of the Cx32 gene using an adeno-associated virus (AAV) vector can improve ...

While researchers have identified more than 90 mutated genes that cause CMT, approximately 50 percent of CMT2 patients do not yet have a definitive genetic diagnosis. Dr. Stephan Züchner at the University of Miami is working to change that.

This CMT Awareness Month, the CMTA is proud to join the MDA as a sponsor of the Hereditary Neuropathy Foundation, who will be hosting the Externally-led Patient-Focused Drug Development Meeting (PFDD).