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…
EN001, a stem cell therapy being developed by Encell, was found to safely ease symptoms of Charcot-Marie-Tooth disease type 1A (CMT1A) in early clinical testing. That’s according to the results of a Phase 1 trial (NCT05333406) involving nine adults with a genetically confirmed diagnosis of CMT1A.
Duplication of the PMP22 gene, the underlying cause of Charcot-Marie-Tooth disease type 1A (CMT1A), disrupts the development of Schwann cells essential to the myelin sheath that protects nerve cells, a study in mouse models and cells derived from patients reports. Specifically, the gene’s duplication dysregulates fat-like lipid metabolism, storage,…
The Hereditary Neuropathy Foundation (HNF) will present a patient-centered Charcot-Marie-Tooth disease (CMT) summit June 7-8 that will address clinical trial readiness. The event will happen at the Loews Coronado Bay Resort, San Diego, and a limited number of discounted rooms are available. Tickets are $200 for patients and…
The CMT Research Foundation is joining Jenny Decker, a nurse with Charcot-Marie-Tooth disease (CMT) who last summer set sail on a solo trip around the world to raise awareness about the disease. The trip, which Decker named Just A Lap, could take about three years. If she succeeds,…
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