News

Endgame campaign surpasses $10M raised for CMT1A treatments

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…

Cochlear implants for CMT may improve hearing, quality of life

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…

CMT1A gene defect disrupts cells crucial for nerve health, study says

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,…

HNF to host CMT disease summit on readiness for clinical trials

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…

Foot surgery eases walking-related pain in CMT: Small study

Foot surgery may help reduce pain during walking and correct the way people with Charcot–Marie–Tooth disease (CMT) stand, a brief report by researchers in Italy suggests. While foot surgery may not address functional limitations like a slow gait and poor balance, it “allows [patients] to wear shoes, and leads…

Pharnext working toward applications for PXT3003 approval

Pharnext is analyzing clinical trial data that could support applications to approve PXT3003, its therapy candidate for people with Charcot-Marie-Tooth type 1A (CMT1A), in the U.S. and Europe. “We remain hopeful of finally being able to bring a medical solution to patients with no treatment,” Hugo Brugière, manager…