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.

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...

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...

A Robust Therapeutic Pipeline for CMT 1A

Dec 19, 2019 | CMT Research Updates, CMTRF Funded Research, Press Releases, Research News

The majority of people with CMT have the 1A subtype which is due to duplication of the peripheral myelin protein 22 (PMP22) gene leading to breakdown of the myelin that surrounds peripheral nerves. With time this loss of myelin causes nerve dysfunction and...