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.

Understanding CMT Genetics

Jun 16, 2020 | About CMT

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,...

Responding to the Pandemic with Persistence and Progress

May 1, 2020 | CMT Research Updates

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...

New Research Project to Advance Treatments for CMT1B

Apr 29, 2020 | CMT Research Updates, CMTRF Funded Research, Drug Development, Research News

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...

Discovery of a new gene found to cause CMT2

Mar 25, 2020 | CMT Research Updates, Research News

CMT Research Foundation Scientific Advisory Board members found a new gene which causes CMT2.

Acceleron discontinues development of ACE-083 for use in CMT

Mar 23, 2020 | CMT Research Updates, Research News

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

Mar 9, 2020 | CMT Research Updates, CMTRF Funded Research, Drug Development

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

Feb 12, 2020 | CMT Research Updates, CMTRF Funded Research, Drug Development, Press Releases, Research News

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

Feb 12, 2020 | CMT Research Updates, CMTRF Funded Research, Drug Development, Press Releases

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

Jan 30, 2020 | CMT Research Updates, CMTRF Funded Research, Drug Development, Press Releases, Research News

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...