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.

Nothing Stops Innovation: Results from BIO 2020

Jun 24, 2020 | CMT Research Updates, Drug Development, News

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 Ends with Me: My Painful Decision Not to Become a Mother

May 7, 2020 | Life with CMT, Press Releases

By: Diane, living with CMT I’d give you my feet if I could. That’s what my strong, stoic mother told me as she struggled to watch me walk as a teenager. I was a natural athlete growing up. I loved running and excelled at it. One day while running, I blew out my...

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...

DTx Pharma Milestone 1 Completed

Apr 8, 2020 | CMTRF Funded Research, Research News

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

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...