News

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).

Regenacy today announced a collaboration with the CMTA to validate the role of HDAC6 in multiple forms of Charcot-Marie-Tooth (CMT) disease and evaluate the efficacy of ricolinostat, a selective HDAC6 inhibitor, in animal models.

“We are excited to establish this partnership with Acceleron as they work to advance their lead neuromuscular therapeutic candidate, ACE-083, through an ongoing Phase 2 trial in patients with CMT,” said CMTA’s CEO Amy Gray.