News

Fellowship awarded to advance research on foot deformities in CMT

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…

CMTA funds to boost gene therapies for less common CMT types

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…

MDA 2025: Conference begins, marking another year of progress

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…

FDA grants orphan drug status to EN001 stem cell therapy for CMT

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…

MDA Clinical & Scientific Conference starts on March 16

Note: This story has been updated March 6, 2025, to correct a quote from Mindy Henderson, MDA vice president of disability outreach and empowerment. The Muscular Dystrophy Association (MDA) is once again hosting its annual gathering, the MDA Clinical & Scientific Conference. The 2025 event will take place…

3D-printed ankle braces may help with balance, foot pain: Study

The use of 3D-printed, customized ankle braces may improve balance and reduce foot pain in people with Charcot-Marie-Tooth (CMT) disease, according to a recent pilot study in New Zealand. Although patients reported variable levels of comfort and satisfaction, they generally said the braces were moderately comfortable, and were moderately…

Children with CMT should be screened for hip dysplasia: Study

A team of U.S. researchers is calling for routine screening for hip dysplasia — when the bones in the hip joint don’t fit together correctly, causing instability — among children with Charcot-Marie-Tooth disease (CMT), after their work revealed it to be a common but underrecognized problem in young people…