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.

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...

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....