Most kinds of CMT come from a problem in a single gene. Repairing that faulty...
CMTA Leading the Way in CMT1A
CMTA is leading a coordinated CMT1A research strategy that connects disease models, drug development, biomarkers, delivery science, and clinical infrastructure to accelerate new treatments.
CMTA-STAR Delivery Science Portfolio: Four Parallel Approaches to Therapeutic Delivery in CMT
CMTA is funding four research projects targeting a fundamental barrier to CMT treatment: getting therapies past the biological defenses protecting peripheral nerves. The $523,000 CMTA-STAR investment supports complementary delivery approaches, including gene editing, RNA medicine, and nanoparticles, across multiple CMT subtypes.
Schwann Cell-Targeted Peptide-LNPs for Delivery of siRNA Against PMP22 in CMT1A
With combined support totaling $102,968 from CMTA, Shark Tooth Bio, and the National Research Council...
Blood–Brain Barrier-Crossing Conjugates for Treating CMT
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.
Can We Fix the Code? A Look at Genetic Therapies for CMT
Can we fix the genes that cause CMT? CMTA’s Dr. Katherine Forsey breaks down the science behind genetic therapies and what’s heading toward clinical trials.
CMTA-STAR Alliance Partner Doses First CMT2S Patient
CMTA-STAR Alliance Partner Doses First CMT2S Patient
CMTA Presents New Genetic Landscape Analysis at The 2025 Annual Meeting of the Peripheral Nerve Society
The Charcot-Marie-Tooth Association (CMTA), the largest philanthropic funder of Charcot-Marie-Tooth (CMT) disease research, presented a...
Accelerating Genetic Therapy for CMT4B1: A CMTA Research Update
With CMTA’s support, researchers at San Raffaele Scientific Institute in Milan, Italy, led by Alessandra...
