News & Stories
See the latest news about CMT drug development and read stories from the CMT community that highlight why we must deliver treatments and cures during our lifetime.
FDA Grants Rare Pediatric Disease Designation to Investigational Therapy for CMT2S
On July 7, 2026, Vanda Pharmaceuticals announced that the FDA has granted Rare Pediatric Disease Designation to VCA-894A, an investigational antisense oligonucleotide (ASO) therapy for Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S). CMT2S is an inherited...
Submit Your Research: 2026 Global CMT Research Convention Accepting Abstracts
Mark your calendar: the 2026 Global CMT Research Convention will take place September 24 to 26 in Cambridge, Massachusetts, at the Royal Sonesta Boston. The event brings together patients, researchers, pharma companies, investors, and regulatory agencies working to...
Alan Jackson’s Final Tour Raises $2.25 Million for CMT Research
Alan Jackson's "Last Call: One More for the Road" tour has officially wrapped, and the number is in: more than $2.25 million was raised for the CMT Research Foundation through ticket sales, matching contributions, and the wave of awareness Alan brought to...
CMT Research Is Moving. Here’s What We’re Working On.
Charcot-Marie-Tooth disease progressively damages the peripheral nerves, causing muscle weakness, loss of sensation, and difficulty walking. There's no approved treatment. That's exactly why the CMT Research Foundation exists, and what we're working to change. ...
Scientific Takeaways from the Peripheral Nerve Society’s Conference
The Peripheral Nerve Society hosted its annual conference in the Netherlands this June, and our research team went to learn about the latest advances, network with field leaders and accelerate progress for CMT treatments and cures. Here are our scientific team's...
One Last Round: Watching Alan Jackson Ride Off Into the Sunset
On June 27, Alan Jackson plays his final concert. After nearly four decades, 35 number one hits, and a catalog that helped define what country music sounds like, he is stepping away from the stage on his own terms. Several years ago, Alan Jackson opened up about...
WEBINAR: Gene Therapies Demystified, Policy, Access, and the Road Ahead
The first session of the CMT Research Foundation’s Gene Therapies Demystified series gave attendees the building blocks: what gene therapy is, why CMT is a compelling target, and what the science suggests about its potential. The second session moved from the...
Project Report: A new way to deliver drugs to nerves in CMT
One of the biggest obstacles in CMT research is not a shortage of potential therapies, but a delivery problem: nerves are extremely difficult to reach through the bloodstream. Dr. Kelly Langert's team at Loyola University Chicago took a creative approach, asking...
A Landmark Review of PMP22: The Protein at the Heart of Many CMT Cases
A major new scientific review published in Chemical Reviews has assembled the most comprehensive account to date of peripheral myelin protein 22 (PMP22), the protein responsible for over half of all CMT diagnoses. Led by Dr. Charles R. Sanders and Dr. Bruce Carter at...
New FDA Draft Guidance Creates Opportunities for Gene Therapy Development
On June 2, 2026, the FDA's Center for Biologics Evaluation and Research released a draft guidance titled Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing. Gene therapy is one of the most promising avenues for...
