Marisa Wexler, MS, senior science writer —

CKD-504, a new compound that works to block the protein HDAC6, was found to restore myelination in cell and mouse models of Charcot-Marie-Tooth disease type 1A (CMT1A) in an early study. Results indicate that CKD-504 – and, more broadly, the strategy of inhibiting HDAC6 – might be…

Mutations in the DST gene that affect the normal functioning of nerve fibers can cause Charcot-Marie-Tooth disease (CMT) type 2, a new study shows. This gene has not previously been implicated in CMT, the researchers said. The study, “Isoform-specific loss of dystonin causes…

The three components of the investigational therapy PXT3003 are all essential to significantly improve features of Charcot-Marie-Tooth disease (CMT) type 1A in cell and animal models, a new study suggests. The findings also indicate that PXT3003 could improve muscle biology in CMT1A by improving the connections between muscles…

A new protocol allows researchers to generate spinal motor neurons derived from Charcot-Marie-Tooth disease (CMT) patients, creating useful models for better understanding the disease and investigating responses to potential therapies. The protocol was described in “Optimized Protocol to Generate Spinal Motor Neuron Cells from Induced Pluripotent Stem…

A newly-opened nonprofit called Shark Tooth Cycling aims to encourage young people in the U.S., especially those affected by Charcot-Marie-Tooth disease (CMT), to get outdoors and ride their bikes. Much like other cycling organizations — such as the diabetes-focused cycling group Team Type 1 — the Pittsburgh, Colorado foundation will work to…

Mutations in the gene HARS1, which are a cause of Charcot-Marie-Tooth disease (CMT), result in reduced protein production in neurons — providing a possible mechanism for how these mutations cause disease — a recent study found. The findings were published in The FEBS Journal, in a study titled…

DTx Pharma has discovered new molecules that could be treatment candidates for Charcot-Marie-Tooth disease (CMT) type 1A (CMT1A), reaching the second milestone of a study designed to find molecules that lower PMP22 levels. The milestone was announced in a press release from the CMT…

Problems with energy production and mitochondria, the powerhouses of cells, may be the underlying cause of an X-linked form of Charcot-Marie-Tooth disease (CMT) called CMTX6, a study in patient-derived motor neurons suggests. The study, “Energy metabolism and mitochondrial defects in X-linked Charcot-Marie-Tooth (CMTX6) iPSC-derived motor…

Mutations that cause Charcot-Marie-Tooth disease type 4B (CMT4B) lead to an increase in the activity of a protein called mTORC1 that is responsible for the problems with myelin that characterize the disease, a study found. The findings suggest that mTORC1 may be a viable therapeutic target in CMT4B, the…

People with Charcot-Marie-Tooth disease (CMT) may have difficulties in understanding speech, even in the absence of hearing loss, caused by problems with the nerve cells that carry signals from the ears to the brain, a study suggests. The study, “Psychoacoustics and neurophysiological auditory processing in patients…