September is CMT Awareness Month, and advocacy organizations are running campaigns to highlight the diverse lived experiences of people with Charcot-Marie-Tooth disease (CMT). According to the Hereditary Neuropathy Foundation (HNF) — whose motto is “CureCMT” — the theme for this year’s awareness month is #ThisIsCMT. The goal, the…
News
A new set of recommendations offers guidance on how to design clinical trials for potential treatments for Charcot-Marie-Tooth disease (CMT), emphasizing a need for flexible approaches that can accommodate the disease’s many subtypes and the importance of incorporating input from the CMT community. The recommendations were developed by a…
A novel treatment being developed by Vanda Pharmaceuticals for a single patient with Charcot-Marie-Tooth disease type 2S (CMT2S) — an ultrarare type of this genetic disorder — has been granted rare pediatric disease designation by the U.S. Food and Drug Administration (FDA). That regulatory status aims to incentivize…
An experimental gene therapy for Charcot-Marie-Tooth disease type 4C (CMT4C) — a rare form of the genetic disease that has muscle weakness as its hallmark symptom — was shown to improve nerve signaling, motor function, and muscle health in a mouse model. Moreover, according to the researchers, the treatment…
A renowned European scientist whose early research led to the discovery of more than 100 genes linked to Charcot-Marie-Tooth disease (CMT) is joining the Charcot-Marie-Tooth Association (CMTA) as a member of its scientific advisory board, the nonprofit announced. Belgian geneticist Vincent Timmerman, PhD, will bring his expertise in…
TVR110, an experimental RNA therapy designed to treat Charcot-Marie-Tooth disease type 1A (CMT1A), showed promising signs of efficacy and an encouraging safety profile in tests done in animals, a study reported. The preclinical work was funded by Armatus Bio, the company developing TVR110. Scientists at Armatus conducted the…
The experimental gene therapy Engensis (VM202) was found to be safe and showed early signs of benefit for people with Charcot-Marie-Tooth disease type 1A (CMT1A), according to results from a small Phase 1/2a clinical trial. Treatment reduced disease severity, mainly by easing sensory neuropathy, or nerve damage affecting sensation, and lessened…
Ignaseclant, NMD Pharma’s experimental oral therapy, may help improve muscle strength and motor function in people with Charcot-Marie-Tooth disease (CMT) by making muscles respond better to weaker nerve signals. That’s according to top-line data from the Phase 2a SYNAPSE-CMT trial (NCT06482437), which also showed consistency in…
Adults with Charcot-Marie-Tooth disease (CMT) say physical symptoms, especially fatigue, along with challenges related to grocery shopping and limited lifestyle-focused guidance from healthcare professionals, can make healthy eating difficult. That’s according to a recent study that also identified factors that helped patients maintain healthier eating habits. These included recognizing…
A Charcot-Marie-Tooth (CMT) patient advocate who heads a nonprofit dedicated to improving the lives of people affected by CMT and related disorders has been selected as this year’s recipient of a key award from the Muscular Dystrophy Association (MDA). Allison Moore, the founder and CEO of the Hereditary Neuropathy Foundation…
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