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...
By: Susan Ruediger, CEO, CMT Research Foundation Our lives have changed so much in just eight short weeks. As I’ve spoken with members of our CMT community, the fear and uncertainty we feel as people who are considered “vulnerable” are palatable. “It takes me twice as...
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...
CMT Research Foundation Scientific Advisory Board members found a new gene which causes CMT2.
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....
There are efforts underway to develop gene therapies for several of the CMT types 2 and 4.
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....
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...
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...
Using Biomarkers to Measure Effective Treatment Options for CMT
Important Research News Announcements