Cycle 4C - CMT4C
disease, a degenerative disease of the peripheral nerves. It has
affected her ability to walk, especially after childbirth, but she works
very hard to keep moving and maintain her quality of life.
caregivers, along with the CMTA, to build a patient community and work
towards a cure. We are in direct contact with researchers all around the
world and have made amazing progress.
We currently have a number of priorities for CMT4C. There is an effort to dose a limited number of patients with an experimental gene therapy that could
offer a cure for the genetic disease. We are hoping to launch a natural
history study that will help us to understand the progression of the
disease and how to measure if a potential therapy is working. And there
are multiple researchers involved in efforts to test existing drugs
against cells and, eventually, mouse models to see if any might have an
effect.
As a member of our team, you can donate or raise money from friends and family. Everything you raise will go into the "Foresee the End of 4C" fund and is earmarked for CMT4C research.
Stay Connected