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.
The Full Agenda: 2026 Global CMT Research Convention
Charcot-Marie-Tooth disease drug development is at a pivotal moment. More active drug trials are underway than ever before, and a growing community of researchers, companies, investors, and patients is working toward treatments. The Global CMT Research...
WEBINAR: Behind the Scenes, 2026 Global CMT Research Convention Preview
Charcot-Marie-Tooth disease drug development is at a pivotal moment, with more active drug trials underway than ever before and a growing community of researchers, companies, investors, and patients working toward treatments. The Global CMT Research Convention exists...
Taking Aim at CMT1J with a genetic medicine
The CMT Research Foundation, in partnership with the 1J Foundation, has funded a new study at the University of Miami led by Dr. Stephan Züchner to evaluate a newly-identified genetic variant linked to Charcot-Marie-Tooth disease type 1J and to test whether a targeted...
Why You Should Be in the Room: Dr. Anna Greka to Keynote Global CMT Research Convention
Don't miss Friday's keynote at the Global CMT Research Convention (September 24 to 26, 2026, Cambridge, MA), featuring Dr. Anna Greka, founder of the Ladders to Cures (L2C) Scientific Accelerator at the Broad Institute of MIT and Harvard. Rare genetic diseases are...
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. ...
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...
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...
