News

Oryzon picks HDAC6 inhibitor to develop as potential CMT treatment

Oryzon Genomics has chosen ORY-4001, a selective histone deacetylase 6 (HDAC6) inhibitor, for clinical development as a potential treatment for Charcot-Marie-Tooth (CMT) disease and other neurological conditions. HDAC6 enzyme inhibitors have shown promise for CMT, amyotrophic lateral sclerosis, and other neurological diseases, Oryzon states. ORY-4001 will now…

Measure of myelin damage may aid demyelinating CMT diagnosis

A measure of the distribution of demyelination — myelin loss — may improve the diagnosis of demyelinating Charcot-Marie-Tooth disease (CMT) and better distinguish it from a related rare disease, a study in Japan found. Specifically, the compound muscle action potential (CMAP) duration ratio was significantly lower in people with CMT…

Electrical Stimulation Lowers Protein Clumps, Aids Myelin: CMT1A Model

Electrical stimulation reduced toxic PMP22 protein clumping and promoted myelin-making activity in a cell model of Charcot-Marie-Tooth disease type 1A (CMT1A), a new study reports. “We expect that the findings … will play a significant role in the potential clinical translation of an electroceutical [electricity-based] treatment for CMT1A disease,”…