News

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…

An orthopedic surgeon at the University of Iowa has been awarded the 2025 Inherited Neuropathy Fellowship to advance her research on foot mobility and intervention in people with Charcot-Marie-Tooth (CMT) disease. The recipient of this year’s fellowship is Bopha Chrea, MD. Chrea’s work focuses on the effective use of…

The Charcot-Marie-Tooth Association (CMTA) is funding two research projects to accelerate the development of genetic therapies for less common forms of Charcot-Marie-Tooth disease (CMT), CMT types 2A and 4B1. The investment is part of the association’s Strategy to Accelerate Research program, known as CMTA-STAR. One project, supported…

Wearable sensors used in the home effectively captured data on physical function that correlated with measures of disease severity taken in the clinic for adults with Charcot-Marie-Tooth disease (CMT), data show. Such remote monitoring tools can aid in understanding how CMT affects patients in daily life, and may supplement…

The Muscular Dystrophy Association‘s (MDA) annual Clinical & Scientific Conference kicked off over the weekend, bringing together patients, caregivers, researchers, clinicians, academics, advocates, and industry leaders to discuss the latest in science and care related to a range of neuromuscular diseases. This year’s conference will bring together hundreds…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Encell’s EN001, an investigational stem cell therapy for Charcot-Marie-Tooth disease (CMT). This designation is intended to accelerate the development of therapies designed to prevent, diagnose, or treat rare diseases…