- A Cure for Caroline and Other CMT StoriesCaroline’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!Read more ...
- Type 1 CMT Gene Therapy Project Now UnderwayMutations 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 ...Read more ...
- CMTA-Funded Research Database Identifies New MutationsWhile 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.Read more ...
- Three Major Patient Advocacy Groups Focused on CMT Research Collaborating on FDA “Voice of the Patient” ReportThis 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).Read more ...
- Regenacy Pharmaceuticals Announces Collaboration with the CMTA to Advance Ricolinostat for the Treatment of Hereditary NeuropathyRegenacy 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.Read more ...