The newly funded study will test whether molecules originally in development as cancer therapeutics can also be used to lower PMP22 and improve nerve health in a model of CMT1A.
Gene Editing for CMT Is Getting Real: The Future Just Shifted Gears
CMTA-supported scientists are using CRISPR to go after the genetic root of CMT1A and CMT1B. Early results are changing what’s possible. See how this research is shifting from bold ideas to future realities.
ACT-CMT: CMT1A Natural History Study, Long-Term Progression, and Biomarkers
With $1.2M in CMTA funding, the ACT-CMT study is tracking long-term CMT1A progression and advancing biomarker development to prepare for future clinical trials.
Targeting the Cell’s Clean-Up System in CMT1
In CMT1A and CMT1B, the cell's protein clean-up system breaks down, allowing harmful proteins to build up and damage peripheral nerves. CMTA-funded researcher Jordan VerPlank, PhD, is working to reactivate that system using medicines already tested in people.
Targeting SREBP Regulation for CMT1A
With CMTA support of $74,000, researchers at the University of Wisconsin-Madison, led by CMTA Strategy...
CMTA’s $450K Investment Targets New Approach to Treatment for Patients with CMT1A and CMT1B
CMTA announced a $450,000 investment supporting research into a cGMP-based therapeutic approach for CMT1A and CMT1B, building on prior CMTA-funded studies.
Development of a Human-Derived Myelin-Containing Organoid as a Reference Model for CMT1A
Researchers led by CMTA-STAR Advisory Board member Vincent Timmerman, PhD, at the University of Antwerp are developing human-derived CMT1A organoids using patient-derived stem cells. This research platform aims to improve understanding of disease mechanisms and provide a standardized model for evaluating potential treatments.
Leading the Way: The CMTA Invests More Than $200K in Cutting-Edge CMT1A Organoid Research
CMTA announced an investment of more than $200,000 to support CMT1A organoid research, establishing standardized models to evaluate therapies targeting CMT1A.
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.
Nanoparticle-Based Gene Delivery to Schwann Cells for Treating CMT Disease
With joint funding of $299,992 from CMTA and the Muscular Dystrophy Association (MDA), researchers at...
