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.
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...
Tiny Movers, Big Clues: Dr. Manor Uses AI to Shed Light on CMT2A
CMTA-funded CMT2A treatment research at UC San Diego is building the tools to develop ASO therapies targeting the faulty MFN2 gene, with a clear path toward treatments.
CMT2A Biomarkers and Natural History
With CMTA support of $572,055, researchers at the Inherited Neuropathy Consortium led by Michael E. Shy, MD, are working to track disease progression and identify biomarkers in CMT2A. By studying blood, skin biopsy, and MRI biomarkers alongside clinical assessments, this project aims to develop critical tools for future clinical trials and advance CMT2A research.
Actio Biosciences – ABS-0871
The Phase I study is a randomized, double-blind, placebo-controlled trial evaluating the safety, tolerability, and...
Solve-CMT Resource: Addressing the Diagnostic Gap and Drug Target Discovery via Advanced Genomics
CMTA-funded researchers at the GENESIS Project Foundation are using advanced genomics and artificial intelligence to accelerate CMT gene discovery, improve genetic diagnosis, and identify new therapeutic targets.
NMD Pharma – SYNAPSE-CMT
This Phase 2a study aims to evaluate the efficacy, safety and tolerability of NMD670 vs...
ASO Approaches for CMT2E
With CMTA support of $225,483, researchers at the University of Miami, led by CMTA Strategy...
CMT2F Biomarkers and Outcome Measures
With CMTA support of $302,071, an international team of researchers from the Inherited Neuropathy Consortium...
2A Biomarkers and Outcome Measures
With CMTA support of $572,055, an international team of researchers from the Inherited Neuropathy Consortium,...
