News & Stories
See the latest news about CMT drug development and read stories from the CMT community that highlight why we must deliver treatments and cures during our lifetime.
Losing My Daughter: What CMT Really Looks Like
By: Michelle Moon Before my daughter Julianna was born, I knew as much about CMT as the average neurologist, which is to say I knew very little. During residency, I met a patient with CMT who was a rock climber. His hand muscles were atrophied, but he spent his...
CMT Ends with Me: My Painful Decision Not to Become a Mother
By: Diane, living with CMT I’d give you my feet if I could. That’s what my strong, stoic mother told me as she struggled to watch me walk as a teenager. I was a natural athlete growing up. I loved running and excelled at it. One day while running, I blew out my...
No One Should Have to Choose Between Motherhood and CMT
I’ve had CMT my entire life. It runs on my dad’s side of the family. My parents knew I had CMT when I was born, but a genetic test confirmed it as a toddler. Early on, CMT affected me most by watching the effects it had on my dad, uncle and grandmother. The memory of...
Creating multiple opportunities for success in CMT 1A
By Grace Pavlath, PhD By now you’ve likely heard that CMTRF is funding three genetic-based projects focused on therapy development for CMT1A: DTx Pharma, Shift Pharma and Cyprus Institute of Neurology and Genetics. Funding multiple complementary approaches gives...

CMT Research Foundation Funds Shift Pharmaceuticals to Advance Search for CMT1A Treatment
Shift to Develop and Analyze Novel Series of Drugs Designed to Control Expression of PMP22 Gene The CMT Research Foundation (CMTRF), a nonprofit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease, today announced it has partnered with...
2019 – A Year of Closing the Gaps in CMT Drug Development
Because of you, the pace of drug development for CMT accelerated! As we close the books on 2019, I am excited to share the progress the CMT Research Foundation has made this year. Because of you, these advancements were possible. We have grown considerably in the...

Because of You
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...

Chief Science Officer comments on recent work
CMT Research Foundation Chief Science Officer Grace Pavlath, PhD comments on recent work by Morelli et al: Allele-specific RNA interference prevents neuropathy in Charcot-Marie-Tooth disease type 2D mouse models Some types of CMT result from the expression of a mutant...
Where is the Progress? The Cure For CMT Calls For Collaboration
More collaboration within the CMT research and patient community would help us get to treatments and a cure much faster. Thomas Durcan, an Assistant Professor for neurodegenerative disorders at McGill University recently wrote a very interesting appeal for...
AcuraStem Achieves First Milestone
Just nine months ago, we announced a milestone-based, research collaboration with AcuraStem to test thousands of compounds aimed at producing effective treatments for CMT2A. We are very pleased to report that AcuraStem has completed phase one of the CMT2A project. To...