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.
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...
CMTA Funds Preclinical Work on Modulating Unfolded Protein Response in CMT1B Mice
CMTA has funded a preclinical study exploring whether modulating the unfolded protein response (UPR) can address disease mechanisms in CMT1B. The project will evaluate activation of the ATF6 pathway in CMT1B mouse models to determine whether this approach can reduce cellular stress linked to myelin dysfunction and inform future therapeutic development.
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...
