With CMTA support of $281,339, researchers at Albany Medical College, led by Sophie Belin, PhD, and Yannick Poitelon, PhD, are exploring the newly discovered TEAD1 pathway in Schwann cells that may regulate PMP22, the gene at the root of CMT1A.
Actio Biosciences – ABS-0871
The Phase I study is a randomized, double-blind, placebo-controlled trial evaluating the safety, tolerability, and...
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.
Targeting SREBP Regulation for CMT1A
With CMTA support of $74,000, researchers at the University of Wisconsin-Madison, led by CMTA Strategy...
Testing New UPR-Targeted Therapies for CMT1B
Researchers at the Ospedale San Raffaele (OSR) Scientific Institute in Milan, Italy, supported by the...
Applied Therapeutics – INSPIRE
This study is designed to assess the efficacy and safety of AT-007 treatment in patients...
