CMT News & Updates
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.
Filter by Category
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…
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…
Read More : Why You Should Be in the Room: Dr. Anna Greka to Keynote Global CMT Research Convention
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…
Read More : FDA Grants Rare Pediatric Disease Designation to Investigational Therapy for CMT2S
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,…
Read More : Submit Your Research: 2026 Global CMT Research Convention Accepting Abstracts
Alan Jackson’s Final Tour Raises $2.25 Million for CMT Research
Alan Jackson - Last Call tour 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…
Read More : Alan Jackson’s Final Tour Raises $2.25 Million for CMT Research
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. We’ve recently been in…
Read More : CMT Research Is Moving. Here’s What We’re Working On.
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…
Read More : One Last Round: Watching Alan Jackson Ride Off Into the Sunset
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…
Read More : Project Report: A new way to deliver drugs to nerves in CMT
A Landmark Review of PMP22: The Protein at the Heart of Many CMT Cases
Dr. Charles R. Sanders featured next to stacks and stacks of papers incorporated into the review. A major new scientific review published in Chemical Reviews has assembled the most comprehensive account to date…
Read More : A Landmark Review of PMP22: The Protein at the Heart of Many CMT Cases
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. …
Read More : New FDA Draft Guidance Creates Opportunities for Gene Therapy Development
