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.
CMTA Invests $141,000 to Study Hearing and Balance to Develop Better Clinical Trial Outcome Measures in Charcot-Marie-Tooth Disease
CMTA is supporting a two-year research project examining hearing and balance signaling to strengthen outcome measures in future CMT clinical trials.
Hearing and Balance Biomarkers for Outcome Measure Development in CMT1A
With CMTA support of $141,000, researchers led by Gabriel Corfas, PhD, at the Kresge Hearing...
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
With CMTA support of $54,051, researchers led by Yizhou Dong, PhD, at the Icahn School...
Preliminary Characterization of STEP Platform for Delivery of Genome Editing Therapy for CMT
With CMTA support of $66,000, researchers led by Jiangbing Zhou, PhD, at Yale University are...
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.