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.
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....
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...
Partnership with DTx Pharma to optimize antisense oligonucleotides as a gene therapy for CMT1A
ATLANTA (December 20, 2019) The CMT Research Foundation (CMTRF), a nonprofit focused solely on delivering treatments and cures for Charcot-Marie-Tooth, today announced it has partnered with DTx Pharma, an RNA medicines company breaking open new therapeutic areas for...
A Robust Therapeutic Pipeline for CMT 1A
The majority of people with CMT have the 1A subtype which is due to duplication of the peripheral myelin protein 22 (PMP22) gene leading to breakdown of the myelin that surrounds peripheral nerves. With time this loss of myelin causes nerve dysfunction and...
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...
Drug Screening in a Dish
At the CMT Research Foundation we are very much interested in using patient-derived stem cells called iPSCs for drug screening in order to develop therapies for CMT. Indeed, one of the first projects we funded was such a drug screening effort for CMT2A at Acurastem....
Gene Editing Used for the First Time in CMT
Gene editing as a therapeutic tool for treating disease has been very much in the news recently. The most commonly used gene editing tool to permanently fix the underlying genetic cause of a disease is CRISPR/Cas9. A recent paper demonstrates the first...
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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