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.

AcuraStem Scientists Demonstrate Positive Early Results for CMT2A Treatment

Nov 10, 2020 | CMT Research Updates, CMTRF Funded Research, Drug Development, Research News

The CMT Research Foundation is currently funding a research project led by AcuraStem aimed at producing effective treatments for CMT2A. Using stem cells derived from adult patients, AcuraStem scientists have tested thousands of compounds for their ability to promote...

CMT Research Foundation Project Shows Progress in a Gene Therapy Approach to Treat CMT1A

Nov 10, 2020 | CMT Research Updates, CMTRF Funded Research, Drug Development, Research News

The CMT Research Foundation is currently funding a research project led by Dr. Kleopas Kleopa and his team at the Cyprus Institute of Neurology & Genetics to study a gene therapy approach to lower levels of PMP22, the gene that causes CMT1A. While no one can...

DTx Pharma Shows Continued Progress in Reducing PMP22 Levels in Animal Models of CMT1A

Nov 10, 2020 | CMT Research Updates, CMTRF Funded Research, Drug Development, Research News

The CMT Research Foundation is currently funding a research project led by DTx Pharma to design genetic therapies for Charcot-Marie-Tooth disease (CMT). By attaching the genetic sequences to molecules called long chain fatty acids, they allow the therapies to target...

Curcumin and CMT: What You Need to Know

Oct 27, 2020 | CMT Research Updates, Drug Development, Research News

“Should I be taking curcumin supplements or eating more turmeric?” Many people with Charcot-Marie-Tooth disease (CMT) have been asking this question after researchers recently published a paper about curcumin and CMT in the journal Free Radical Biology and Medicine. The CMT Research Foundation’s chief scientific officer explores the findings and what it means for people with CMT.

CMT Research Foundation Launches New Research Project to Design Precision Medicine Approach for Charcot-Marie-Tooth Disease

Oct 14, 2020 | CMT Research Updates, CMTRF Funded Research, Drug Development, Research News

In this new CMT Research Foundation-funded project, Drs. Lorson and Garcia at the University of Missouri will develop and test a new gene therapy approach that will both silence the abnormal gene and simultaneously replace it with genetic material that will produce normal protein. The research team will test this approach in an animal model of CMT2E that is already well understood, making it suitable for this proof-of-concept project. If the approach is successful, it could potentially be used for other forms of CMT as well. In fact, it is possible this approach could be used for many CMT-causing mutations that require both silencing of a mutated gene and replacement with normal protein — whether the mutation is currently known or has yet to be discovered. The ability to silence and replace genes, regardless of the specific mutation, is what makes this a precision medicine approach.

CMT Research Foundation Announces New Research to Study Inflammation as Potential Treatment Target for Charcot-Marie-Tooth Disease

Oct 6, 2020 | CMT Research Updates, CMTRF Funded Research, Drug Development, Research News

The CMT Research Foundation is proud to announce a new research collaboration with renowned nerve disease expert Dr. Rudolf Martini of University Hospital Würzburg to investigate whether inhibiting inflammation in the peripheral nerves could reduce symptoms and improve outcomes for patients with Charcot-Marie-Tooth disease Type 1B. Read more about this project and see the four stages in the project.

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...

CMT Genetic Testing: What’s Involved?

Jun 16, 2020 | About CMT

During the past several decades, researchers have discovered more than 100 genetic mutations that cause CMT. In the same time frame, scientists have developed tests that can look at parts of someone’s genetic makeup and identify changes that could cause certain...