CMTA-funded research at Mount Sinai is studying how RNA medicines can be delivered into nerve cells in CMT, overcoming biological barriers that block treatment.
Preliminary Characterization of STEP Platform for Delivery of Genome Editing Therapy for CMT
CMTA-funded Yale researchers are testing STEP, a non-viral delivery system for genome-editing medicines in CMT.
CMTA-STAR Alliance Partner NMD Pharma Shares SYNAPSE-CMT Trial Update
New Phase 2a CMT trial results are in. NMD Pharma shares details.
ACT-CMT: The Largest-Ever Study of CMT Opens [Casting] Call for More Participants
The largest CMT1A study is expanding. New participants needed, including healthy volunteers. You can move this research forward.
Particles on a Mission: Can Nanoparticles Solve One of CMT’s Largest Hurdles?
Can nanoparticles solve one of CMT’s largest hurdles? A team in Cyprus is testing nanoparticle “mail carriers” designed to deliver genetic therapies directly into Schwann cells.
CMT2A in the Lab: A Closer Look at How Treatments Are Tested
What if you could watch CMT2A unfold inside a neuron? CMTA’s new partnership just made it possible, and the early findings are changing the game.
CMT Community Helps Achieve a Major Research Milestone with NMD Pharma
What happens when the CMT community takes action and participates in CMT research? NMD Pharma answers the question.
How the CMT2C Community Is Driving the Path to Treatment
How is the CMT2C community helping move research forward? A CMTA-hosted focus group on how patient voices are shaping the path to a treatment.
Modulating TEAD1 Activity in Charcot-Marie-Tooth Disease Type 1A
With CMTA support of $281,339, researchers at Albany Medical College, led by Sophie Belin, PhD, and Yannick Poitelon, PhD, are exploring the newly discovered TEAD1 pathway in Schwann cells that may regulate PMP22, the gene at the root of CMT1A.
A New Intermediate Type of CMT has Entered the Room: Meet CMTRIE
What does it mean when a nerve conduction pattern falls between demyelinating and axonal CMT? Meet CMTRIE, a new type of CMT caused by mutations in the KCTD11 gene.
