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 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...
CMT Genetic Testing: What’s Involved?
During the past several decades, researchers have discovered more than 100 genetic mutations that cause CMT. In the same time frame, scientists have developed tests that can look at parts of someone’s genetic makeup and identify changes that could cause certain...
Understanding CMT Genetics
CMT is a genetic disorder, meaning it’s caused by a change, or mutation, in your genes. If you have CMT, you might be wondering how you got it. It’s likely that other people in your family have it, too — though this isn’t always the case. To understand how and why,...
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....