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The path to discovering treatments for CMT is anything but a SINGLE path; rather, it is a series of multiple networks running along and intersecting each other, much like the nervous system in which it affects.
Each type and mutation is unique in it’s own way and must be researched accordingly. However, a discovery for one CMT mutation can be a win for many others. Each new puzzle piece provides a clearer picture of CMT as a whole and lays the groundwork for future trials.
Our research pipeline targets the inhibition of axonal degeneration and could potentially work for all type 2 CMT’s. CMT2A is a dominant form of CMT with mutations in the mitofusin (MFN2) gene. HNF has developed a CMT2A mouse model that has been utilized as a tool to test small molecules. One of our most recent funded CMT2A research initiatives was published in. Experimental Neurology in June 2020. 328:113281. doi: 10.1016/j.expneurol.2020.113281. Epub 2020 Mar 5.