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...
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...
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...
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...
Global CMT Research Convention: Highlights to Expect in 2026
The Global CMT Research Convention brings together the full spectrum of the Charcot-Marie-Tooth ecosystem in one place: leading scientists, biotech and pharma companies, investors, regulatory agencies and patients, all focused on accelerating treatments for the most...
Nobody Ran a Single Step at this CMTRF Fundraiser Run
Nearly 200 people gathered at Forgotten Star Brewing in Fridley, Minnesota, for one of the most unique fundraising events around: the Zero K Run, benefiting the CMT Research Foundation. The concept was simple. Participants got the full race-day experience, complete...
