In 2007, HNF developed the Therapeutic Research in Accelerated Discovery (TRIAD) program, a collaborative effort with academia, government and industry, to develop treatments for CMT. Currently TRIAD involves many groups that span the drug discovery, drug development, and diagnostics continuum. The following briefly summarizes who they are and what they are contributing through TRIAD to help bring treatments to those living with CMT.
Developed CMT2A (MFN2 mutation) mouse model
Refined the MFN2 mouse model for distribution among the CMT research community
CMT1A assay (PMP22 mutation) development
“Proof of concept” studies on an in vivo mouse model with CMT2A
Low content screens in zebrafish with CMT2A (MFN2)
Without patient participants, clinical trials are not possible. Thus, it is crucial that clinical registries be developed to provide the data and potential participants for researchers who are working on treatments for inherited neuropathies. Our registry, in comparison to other contact registries, collects essential clinical and genetic information on patients diagnosed with the various forms of hereditary neuropathies in order to advance therapy development for these debilitating disorders. By completing your profile, your information will be utilized to advance research and clinical trials that could lead to future treatments and cures.