- 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 ...
- CMTA Announces Strategic Partnership with Acceleron to Advance CMT Treatment Options“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.Read more ...
- Toxic Medications List For Patients with Charcot-Marie-Tooth DiseaseWhat is a Neurotoxic Medication? Neurotoxicity is the capacity of chemical or biologic agents to induce functional or structural changes in the nervous system resulting in tissue injury. There are many substances that produce damage to normal peripheral nerves, including heavy ...Read more ...