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...
Augustine Therapeutics – AGT-100216
The Phase I trial will focus on safety, tolerability, and pharmacokinetics (how the drug moves...
Long Read Sequencing in Undiagnosed Axonal CMT Patients
The answer is hiding in your DNA. For some, it hasn’t been found yet. This CMTA-funded study uses long-read sequencing to uncover genetic causes of CMT that standard tests may miss.
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.
Solve-CMT Resource: Addressing the Diagnostic Gap and Drug Target Discovery via Advanced Genomics
What happens when CMTA-supported scientists use AI to analyze thousands of CMT genomes for undiscovered genes?
NMD Pharma – SYNAPSE-CMT
This Phase 2a study aims to evaluate the efficacy, safety and tolerability of NMD670 vs...
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.
