News

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…

Blocking a protein called CMTM6 in myelin-producing cells increases the thickness of nerve fibers and the speed at which electrical signals travel along their length, a mouse study has found. Based on these findings, researchers argued that blocking the activity of this protein may be a valuable therapeutic approach…

This October, in observance of Charcot-Marie-Tooth disease (CMT) awareness month in Europe, the CMT&Me study is launching an international competition aimed at informing people about this group of inherited disorders, and raising funds for research, according to a press release. The competition only requires that patients in…

The National Institute of Neurological Disorders and Stroke, part of the National Institutes of Health (NIH), has awarded DTx Pharma a funding grant to advance development of promising new therapeutic candidates for Charcot-Marie-Tooth (CMT) type 1A (CMT1A) disease. The therapeutic candidates are specially designed RNA oligonucleotide…

Three new genetic mutations in the GJB1 gene have been found inn Chinese patients that can cause X-linked Charcot-Marie-Tooth (CMTX1) disease, expanding the diversity of mutations causing the condition, according to a study. While the three mutations were all in coding regions, some previously known mutations were…

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…

A new study is recruiting adults with Charcot-Marie-Tooth disease (CMT) type 1A to find better methods for measuring disease progression in this patient population, the Hereditary Neuropathy Foundation announced in a press release. Named ACT-CMT, for Accelerate Clinical Trials in Charcot-Marie-Tooth Disease, the international…

Pharnext and the Charcot-Marie-Tooth Association (CMTA) have partnered to find new biomarkers associated with Charcot-Marie-Tooth disease type 1A (CMT1A), the most common subtype of the disease, according to a press release. Its goal is to identify and validate CMT1A biomarkers that signal responses to treatment, and…

This summer, the Charcot-Marie-Tooth Association (CMTA) turned its annual Camp Footprint for children and teens with CMT disease into a virtual experience. The unique initiative, which was launched by the CMTA in 2016, seeks to provide a safe sleepaway camp environment where children and adolescents with…