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.

WATCH: When Can Patients Expect Treatments for CMT?

Aug 31, 2020 | Drug Development, Life with CMT

In this first episode of the CMT Research Foundation’s new video series that asks and answers the toughest questions about CMT treatments, leading research experts will have you on the edge of your seat as they share when patients can expect treatments for CMT and how they can participate in accelerating progress.

5 Ways CMT Patients Can Take Action to Accelerate Drug Development

Aug 24, 2020 | Drug Development, Life with CMT

Unlike other neuromuscular diseases, CMT patients have no approved treatments. See how we’re applying breakthroughs in SMA to advance CMT drug development.

How Breakthroughs in Other Diseases Can Impact CMT

Jul 30, 2020 | CMTRF Funded Research, Drug Development

Unlike other neuromuscular diseases, CMT patients have no approved treatments. See how we’re applying breakthroughs in SMA to advance CMT drug development.

Nothing Mild About CMT

Jun 26, 2020 | CMT Research Updates, Drug Development

By: Susan Ruediger, CEO, CMT Research Foundation When I talk to individuals with CMT or share my personal experience about living with the disease, the responses I hear can be heartbreaking, sometimes even infuriating. “At least you don’t have cancer.” “You don’t look...

DTx Pharma Achieves Second Milestone in Study to Advance Treatments for CMT1A

Jun 26, 2020 | CMT Research Updates, CMTRF Funded Research, Drug Development, Research News

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

Jun 24, 2020 | CMT Research Updates, Drug Development, Press Releases

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...

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...

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, 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...