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...
Responding to the Pandemic with Persistence and Progress
By: Susan Ruediger, CEO, CMT Research Foundation Our lives have changed so much in just eight short weeks. As I’ve spoken with members of our CMT community, the fear and uncertainty we feel as people who are considered “vulnerable” are palatable. “It takes me twice as...
New Research Project to Advance Treatments for CMT1B
The CMT Research Foundation is pleased to announce a new partnership with The Jackson Laboratory that will help us better understand CMT1B and potentially lead to the discovery of treatments. CMT1B is the third most common form of CMT. It’s caused by a defect in the...
DTx Pharma Milestone 1 Completed
Our partners at DTx Pharma continue to make great progress on suppressing the overexpression of PMP22, which causes CMT1A. DTx Pharma’s proprietary technology has the potential to render RNA-based therapeutics such as ASO and siRNA more potent, safer and longer-acting...
Discovery of a new gene found to cause CMT2
CMT Research Foundation Scientific Advisory Board members found a new gene which causes CMT2.
Acceleron discontinues development of ACE-083 for use in CMT
Acceleron recently announced that treatment with ACE-083 in patients with CMT did not demonstrate functional improvement in the Phase 2 trial. ACE-083 is a drug which is injected locally into a muscle in order to stimulate growth of the injected muscle....
Gene Therapy for Axonal Neuropathies
There are efforts underway to develop gene therapies for several of the CMT types 2 and 4.
Milestone 2 Completed for CMT1A Project from Cyprus Institute of Neurology and Genetics
In February 2020, Dr. Kleopa completed milestone 2 of the 24-month project and the results continue to be encouraging. The goal during this 6-12 month period of the project was to further optimize the amount of PMP22 gene silencing in a mouse model of CMT1A. Dr....
Important Research News Announcements
- The Role of ESCRTs in Regulating Nervous System Function November 30, 2024
- Genomic Studies in Charcot-Marie-Tooth Disease July 31, 2024
- Charcot-Marie-tooth disease type 2A: An update on pathogenesis and therapeutic perspectives March 5, 2024
- Accelerate Clinical Trials in CMT (ACTCMT) Study March 1, 2024
- Creation of a Schwann Cell Gene Regulatory Network February 29, 2024
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