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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…

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…

The CMT Association is investing $423,000 in the Genesis database to help improve diagnoses of Charcot-Marie-Tooth disease (CMT). The investment, part of the association’s Strategy to Accelerate Research program known as CMTA-STAR, is intended to help researchers in identifying additional genes whose mutations are causes of CMT.

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to investigational oral therapy NMD670 to treat Charcot-Marie-Tooth disease (CMT). Orphan drug designation is given to therapies designed to prevent, diagnose, or treat rare diseases, defined as one affecting fewer than 200,000 people in the U.S. The…

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…

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…

The CMT Research Foundation (CMTRF) has raised more than its $10 million goal for the Endgame campaign to support research toward treatments for Charcot-Marie-Tooth disease type 1A (CMT1A). The foundation announced the results at the annual Global CMT Research Convention, which took place Sept. 26-28 in…

For Charcot-Marie-Tooth (CMT) disease Awareness Month this September, supporters are taking to social media to put a spotlight on the relatively unknown neuromuscular disease. CMT is a group of diseases affecting the network of nerves throughout the body that are involved in sensation and movement. It is one of…

Cochlear implants are a safe and reliable way to improve hearing, speech, and quality of life for people with Charcot-Marie-Tooth disease (CMT), according to a case series. This study provides “more evidence supporting the potential benefit of cochlear implantation among CMT patients,” researchers wrote. “The present analysis is also…