This year I am fortunate to celebrate the holidays with my family. As we prepared to gather three generations for Thanksgiving, my sister and I made an unusual stop: we rented a ramp so my mother could get into my sister’s home.
My mom’s CMT has progressed enough that, at 74 years old, she needs a walker and AFOs on both legs. Because she is in chronic pain, she doesn’t move well or frequently.
I come from five generations of CMT and now I’m watching my cousins’ kids suffer from the early signs: high arches, painful shoes, and twisted ankles. How many generations have to live like this before we end CMT?
It’s a question I get a lot from the patient community, and quite frankly, I don’t have a definitive answer. Because of You, I have reason to hope.
The power of the patient is undeniable. Because of you and broader CMT community, the following research milestones have been reached:
- We’ve identified over 100 genes which cause CMT.
- We know why CMT manifests in over 80% of patients and how to target the problem to change the course of the disease.
- We are expanding the number of researchers dedicated to CMT.
- Most importantly, therapies are being approved for other diseases similar to CMT, paving the way toward approval of treatments.
Key obstacles remain on our journey to treatments and cures for CMT. Because of you, we’ll work to remove them:
- Adequate delivery of potential treatments into the peripheral nervous system is complex.
- Models used in pre-clinical experiments need to be refined to demonstrate better efficacy of potential treatments in people.
- Methods to prevent nerve degeneration aren’t well researched.
This holiday season, unleash the power of your gift. Share our year end campaign and make a donation, large or small, to support our mission. All donations before December 31st will be matched 100% up to $150,000.
We won’t give up until we stop CMT. We’ll get there because of you.
by Susan Ruediger, co-founder and CEO of the CMT Research Foundation