News

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…

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,”…

Practicing sports may enhance life quality and lessen neuropathic pain in people with Charcot-Marie-Tooth 1A (CMT1A), according to a study conducted in Italy. Patients performing sports regularly reported significantly better general health, social function, and mental health. “These results encourage the prescription of…

When Kenneth Raymond was diagnosed with Charcot-Marie-Tooth (CMT) disease subtype 1A more than 20 years ago, there was little information about the disease that a layman could read and understand. So he set out to remedy that. In 2020, Raymond launched a website called the Cryptid Sloth (now known…

Inherited variants in the PSAT1 gene caused Charcot-Marie-Tooth (CMT) disease in two adolescents, as detailed in a case study and reportedly for a first time. These cases are unusual, given that PSAT1 variants are known to cause severe abnormalities in the central nervous system (CNS), or the brain and…