News

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Vanda Pharmaceuticals’ VCA-894A for treating Charcot-Marie-Tooth disease type 2S (CMT2S). An orphan drug designation seeks to encourage the development of therapies for rare diseases — those affecting fewer than 200,000 people in the U.S. — through…

Neuropathic pain due to damage or impairment in the nervous system is common with Charcot-Marie-Tooth disease and linked with heightened social isolation and poorer life satisfaction, according to data from the recent American Academy of Neurology (AAN) annual meeting. The findings suggest “prioritized treatment of neuropathic pain represents a…

A 52-year-old man was diagnosed with Charcot-Marie-Tooth disease type 1 subtype A (CMT1A), after presenting a history of muscle weakness in his legs, foot deformities, and walking impairments. The report illustrates how identifying relatives with similar symptoms may lead to an earlier referral in subsequent CMT cases in the…

The CMT Research Foundation (CMTRF) has invested in a project at Vanderbilt University School of Medicine Basic Sciences for potential new treatments for Charcot-Marie-Tooth disease type 1A (CMT1A). The project will be conducted by Charles Sanders, PhD, a biochemistry and medicine professor and vice dean at Vanderbilt. Sanders’…

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…

The CMT Research Foundation (CMTRF) is joining forces with Augustine Therapeutics to accelerate research into a small molecule therapy for Charcot-Marie-Tooth disease (CMT), with the goal of moving it into clinical testing. With CMTRF and past financial support, Augustine will conduct further studies in its lead, third-generation…

The CMT Research Foundation (CMTRF) is joining forces with Nanite, a biotechnology company, to work toward the development of new gene therapies for Charcot-Marie-Tooth disease (CMT). Nanite will use its artificial intelligence platform to design delivery vehicles that can get genetic medicines to certain cells in the…

AT-007, an experimental treatment from Applied Therapeutics, was found to more than halve the levels of a sugar alcohol called sorbitol in people with SORD deficiency, a type of Charcot-Marie-Tooth disease (CMT) in which too much sorbitol causes nerve damage or neuropathy. Those findings come from a new…

A particular mutation in the PMP22 gene, called T118M, is a risk factor for developing a mild form of Charcot-Marie-Tooth disease (CMT) — but it doesn’t always cause the rare disorder, according to a new study. A series of experiments in cell cultures revealed possible reasons why T118M leads to…

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…