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.

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....

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....

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...

Importance of Biomarkers in CMT

Jan 21, 2020 | CMT Research Updates, Drug Development, Press Releases, Research News

Using Biomarkers to Measure Effective Treatment Options for CMT

Targeting Axon Degeneration – a treatment for all types of CMT?

Jan 7, 2020 | CMT Research Updates, Research News

Preventing axon degeneration could stop the progression of CMT. Read about the efforts by pharma to stop axon degeneration.

Are We Prepared for Clinical Trials in Charcot-Marie-Tooth?

Jan 7, 2020 | CMT Research Updates, Press Releases, Research News

A Review of Clinical Trial Readiness for CMT BY CMT RESEARCH FOUNDATION'S SCIENTIFIC ADVISORY BOARD MEMBER DR. ALEXANDER ROSSOR Read the full paper In this issue of Brain Research, Mike Shy, Mary Reilly and I summarize the current array of outcome measures for...

Partnership with DTx Pharma to optimize antisense oligonucleotides as a gene therapy for CMT1A

Dec 20, 2019 | CMT Research Updates, CMTRF Funded Research, Press Releases, Research News

ATLANTA (December 20, 2019) The CMT Research Foundation (CMTRF), a nonprofit focused solely on delivering treatments and cures for Charcot-Marie-Tooth, today announced it has partnered with DTx Pharma, an RNA medicines company breaking open new therapeutic areas for...