Unlike other neuromuscular diseases, CMT patients have no approved treatments. See how we’re applying breakthroughs in SMA to advance CMT drug development.
How Breakthroughs in Other Diseases Can Impact CMT
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DTx Pharma Achieves Second Milestone in Study to Advance Treatments for CMT1A
The CMT Research Foundation is pleased to announce that our partner DTx Pharma just completed the second milestone in a study we’re funding which uses their proprietary technology to suppress the overexpression of PMP22, the protein that causes CMT1A. Their work has...
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...
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...
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...