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....
Susan B. Canavari Joins CMT Research Foundation Board of Directors
Deeply Experienced Brand Marketer to Help Increase Awareness of CMT and Accelerate Research ATLANTA (September 21, 2020) The CMT Research Foundation, a nonprofit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease during our lifetime,...
CMT Patients & Families Need More Than Awareness
It has been more than 130 years since CMT was first discovered, yet there are still no treatments or cures. This CMT Awareness Month, we need more than awareness. We need action. Here’s what we can do together to move the needle now.
CMT Research Foundation Adds New Chief Operating Officer and Chief Scientific Officer
Meet the CMT Research Foundation’s new Chief Operating Officer Erich Fasnacht. See how he plans to increase the number and scale of CMT research projects.
Meet the CMT Research Foundation’s New Chief Operating Officer Erich Fasnacht
Meet the CMT Research Foundation’s new Chief Operating Officer Erich Fasnacht. See how he plans to increase the number and scale of CMT research projects.
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...
Nothing Stops Innovation: Results from BIO 2020
By: Susan Ruediger, CEO, CMT Research Foundation “We’re not currently working in CMT, but we know we should be.” “We think our drug could work for CMT, but it has never been tested.” “CMT is definitely on our interest list for expansion, but we need to learn more...
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...
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