News

Pharnext and the Charcot-Marie-Tooth Association (CMTA) have partnered to find new biomarkers associated with Charcot-Marie-Tooth disease type 1A (CMT1A), the most common subtype of the disease, according to a press release. Its goal is to identify and validate CMT1A biomarkers that signal responses to treatment, and…

This summer, the Charcot-Marie-Tooth Association (CMTA) turned its annual Camp Footprint for children and teens with CMT disease into a virtual experience. The unique initiative, which was launched by the CMTA in 2016, seeks to provide a safe sleepaway camp environment where children and adolescents with…

The CMT Research Foundation (CMTRF) wants to turn September from Awareness Month into “CMT Action Month.” This year, the foundation is seeking to turn awareness of Charcot-Marie-Tooth (CMT) disease into action through useful and informative videos, personal fundraising pages, and a “double-your-donation” fundraising campaign. Launched in 2018…

From participating in a virtual dog walk, to switching out Facebook photo frames, advocates are set to mark Charcot-Marie-Tooth (CMT) Awareness Month, observed every September. Awareness and education are crucial to increasing the recognition, diagnosis, understanding, and management of CMT, a group of inherited peripheral nervous…

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

The extra copy of the PMP22 gene that causes Charcot-Marie-Tooth disease type 1A (CMT1A) leads to a potentially toxic buildup of the PMP22 protein inside cells because the excess protein cannot be folded properly and is not transported to its rightful place in the cell membrane, according…

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience.  Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children with…

CMT Australia, a charity association aiming to raise awareness and empower people with Charcot-Marie-Tooth disease (CMT) to live their lives to the fullest, has received $10,000 to support the organization’s CMT Aussie Kids youth program. The Commonwealth Bank Community Grant will support activities for younger CMT patients, as…

The three components of the investigational therapy PXT3003 are all essential to significantly improve features of Charcot-Marie-Tooth disease (CMT) type 1A in cell and animal models, a new study suggests. The findings also indicate that PXT3003 could improve muscle biology in CMT1A by improving the connections between muscles…