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.
CMT Research Foundation Launches New Research Project to Design Precision Medicine Approach for Charcot-Marie-Tooth Disease
In this new CMT Research Foundation-funded project, Drs. Lorson and Garcia at the University of Missouri will develop and test a new gene therapy approach that will both silence the abnormal gene and simultaneously replace it with genetic material that will produce normal protein. The research team will test this approach in an animal model of CMT2E that is already well understood, making it suitable for this proof-of-concept project. If the approach is successful, it could potentially be used for other forms of CMT as well. In fact, it is possible this approach could be used for many CMT-causing mutations that require both silencing of a mutated gene and replacement with normal protein — whether the mutation is currently known or has yet to be discovered. The ability to silence and replace genes, regardless of the specific mutation, is what makes this a precision medicine approach.
CMT Research Foundation Announces New Research to Study Inflammation as Potential Treatment Target for Charcot-Marie-Tooth Disease
The CMT Research Foundation is proud to announce a new research collaboration with renowned nerve disease expert Dr. Rudolf Martini of University Hospital Würzburg to investigate whether inhibiting inflammation in the peripheral nerves could reduce symptoms and improve outcomes for patients with Charcot-Marie-Tooth disease Type 1B. Read more about this project and see the four stages in the project.
CMT Research Foundation Partner DTx Pharma Wins Grant from National Institutes of Health for CMT1A Gene Therapy Program
CMT Research Foundation partner DTx Pharma, Inc., has won a grant from the National Institutes of Health (NIH) to further fund its work on CMT. The investment is significant because the CMT Research Foundation gave DTx its first CMT-related grant just nine months ago....
How Breakthroughs in Other Diseases Can Impact CMT
Unlike other neuromuscular diseases, CMT patients have no approved treatments. See how we’re applying breakthroughs in SMA to advance CMT drug development.
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...
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.