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
Apply Now: Emerging Researcher Forum at the 2026 Global CMT Research Convention
The Emerging Researcher Forum is a closed, invite-only session held the morning of Day One of the 2026 Global CMT Research Convention, ahead of the main Convention program that kicks off that afternoon. Eligible forum…
Read More : Apply Now: Emerging Researcher Forum at the 2026 Global CMT Research Convention
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…
Read More : The Full Agenda: 2026 Global CMT Research Convention
WEBINAR: Behind the Scenes, 2026 Global CMT Research Convention Preview
https://youtu.be/sjKZ7Przv1M 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…
Read More : WEBINAR: Behind the Scenes, 2026 Global CMT Research Convention Preview
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…
Introducing the CMTRF Journal Club: A New Interactive Series for CMT Researchers
The CMT Research Foundation is launching a new Journal Club webinar series designed specifically for researchers working in the CMT space. Each session will spotlight a recent, high-impact publication, with a short…
Read More : Introducing the CMTRF Journal Club: A New Interactive Series for CMT Researchers
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.
