News

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.

Amy Keller had always known about CMT as it runs in her family. But, as a healthy, active child who grew up into a symptom-free adult, it had never crossed her mind that she, too, would one day receive that ...

The CMTA announces the funding of a clinical research study that will look at pulmonary function for people who have CMT Type 2A, with the aim of developing a night-time breathing treatment similar to CPAP for CMT2A patients.

Completely funded by the CMTA, the 18-month project headed by Dr. Kleopas A. Kleopa of the Cyprus Institute of Neurology and Genetics will explore the possibility of using gene editing to treat CMT4C.