CMT looks different from person to person, even within the same family. A newly published review by CMTA-STAR Advisory Board members and leading CMT experts brings together what is currently known about CMT variability, genetics, and disease mechanisms into a single, organized reference.
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.
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...
CMTA Addresses the Critical Delivery Challenge Underlying CMT Treatment Development through Four Key Investments
CMTA announced $523,000 in funding for four research projects focused on solving one of the biggest challenges in CMT treatment development: delivering therapies to peripheral nerve cells. By investing in genome-editing transport, blood-nerve barrier crossing, Schwann cell targeting, and nanoparticle systems, CMTA is working to ensure treatments can reach the cells where they are needed.
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.
When Early Testing Shows a Program Is Worth Continuing
Some research ideas reach a point where early testing answers an important question: Is this work worth continuing? For EverTree Bio, that moment came through early work using the CMTA Preclinical Toolbox.
