News

Ignaseclant, NMD Pharma’s experimental oral therapy, may help improve muscle strength and motor function in people with Charcot-Marie-Tooth disease (CMT) by making muscles respond better to weaker nerve signals. That’s according to top-line data from the Phase 2a SYNAPSE-CMT trial (NCT06482437), which also showed consistency in…

Adults with Charcot-Marie-Tooth disease (CMT) say physical symptoms, especially fatigue, along with challenges related to grocery shopping and limited lifestyle-focused guidance from healthcare professionals, can make healthy eating difficult. That’s according to a recent study that also identified factors that helped patients maintain healthier eating habits. These included recognizing…

A Charcot-Marie-Tooth (CMT) patient advocate who heads a nonprofit dedicated to improving the lives of people affected by CMT and related disorders has been selected as this year’s recipient of a key award from the Muscular Dystrophy Association (MDA). Allison Moore, the founder and CEO of the Hereditary Neuropathy Foundation…

A novel mutation in the MPZ gene was behind an unusual case of Charcot-Marie-Tooth disease (CMT), according to a research report. Understanding the underlying genetic biology of CMT, especially when the disease presents in unexpected ways, will be essential for developing targeted treatments, researchers said in the study, “…

Surgery to correct ankle and foot deformities enabled adults with Charcot-Marie-Tooth disease (CMT) to achieve certain personal goals, such as walking barefoot or walking on uneven ground with shoes on, a new study revealed. Undergoing a surgical correction also significantly improved balance among CMT patients, the researchers noted —…

Patient advocacy organizations CureCMT4J and the CMT Research Foundation (CMTRF) are teaming up with Elpida Therapeutics for a clinical trial of ELP-02, an experimental gene therapy for CMT4J, an ultra-rare form of Charcot-Marie-Tooth disease (CMT). “Our collaboration with Elpida Therapeutics and CMTRF enables us to move…

September is CMT Awareness Month, and organizations are working to increase visibility of Charcot-Marie-Tooth (CMT) disease — a disease that often goes unseen. “CMT is not invisible because it is rare. It is invisible because no one talks about it,” the Charcot-Marie-Tooth Association stated on its awareness month…

The Zeus KAFO, a new device from Icarus Medical Innovations, aims to support mobility in people with lower limb weakness, instability, or paralysis due to conditions such as Charcot-Marie-Tooth disease (CMT). The Zeus device is a knee-ankle-foot orthosis (KAFO), meaning it’s worn from the…

Cure CMT is partnering with Tamarack Habilitation Technologies to expand access to advanced orthotic devices — used to correct and support bone and muscle — for people with Charcot-Marie-Tooth…

Mutations in the GJB1 gene affecting transmembrane, or cell membrane-spanning, regions of the connexin-32 protein are associated with more severe disease in people with X-linked Charcot-Marie-Tooth disease type 1 (CMTX1). That’s according to a study in France, which also demonstrated that patients with mutations affecting other protein regions usually…