CMTA is leading a coordinated CMT1A research strategy that connects disease models, drug development, biomarkers, delivery science, and clinical infrastructure to accelerate new treatments.
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.
Allele-Specific Silencing of MFN2 to Treat Peripheral Neuropathy
With CMTA support of $300,000, CMTA Strategy To Accelerate Research (CMTA-STAR) Advisory Board member Bruce...
CMTA’s $300K Investment in CRISPR for CMT2A
The Charcot-Marie-Tooth Association (CMTA) is investing $300,000 in a groundbreaking gene editing research project at...
CMTA’s $300K Investment in CRISPR for CMT2A
CMTA announced a $300,000 CMTA-STAR investment supporting CRISPR-based gene editing research aimed at selectively silencing disease-causing MFN2 mutations in CMT2A.
Gene Editing Strategies for Demyelinating CMT
CMTA-supported researchers are advancing CRISPR-based gene editing for CMT1A and CMT1B, laying the foundation for mutation-specific therapies.
CMTA Seed Money Draws NIH Support for Type 2 Gene Editing, Neurofilament Projects
CMTA seed funding has helped secure additional NIH support for two CMT type 2 research projects focused on gene editing and neurofilament restoration. The NIH awards build on CMTA-funded pilot work and reflect the role of early philanthropic investment in advancing translational CMT research toward therapeutic development.
