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.

DTx Pharma, Inc. Completes CMT Research Foundation-Funded Project

Aug 27, 2021 | CMT Research Updates, CMTRF Funded Research, Drug Development

The CMT Research Foundation is pleased to announce that DTx Pharma, Inc. (DTx) has successfully completed their CMT Research Foundation-funded RNA-based therapy project. With funding from the CMT Research Foundation, DTx designed and tested a library of novel...

CMT Research Foundation Targets CMT1X in Partnership with University of Illinois Chicago

Jul 8, 2021 | CMT Research Updates, CMTRF Funded Research, Drug Development, News, Research News

The CMT Research Foundation is partnering with the University of Illinois Chicago to test a potential therapeutic for CMT1X, the second most common form of CMT, behind only CMT1A in prevalence. CMT is one of the most common inherited neurological disorders, affecting...

CMT Research Foundation partners with Biotechnology Company AcuraStem and CMT Researcher Alessandra Bolino to test new drug in CMT4B1

Jun 27, 2021 | About CMT, CMT Research Updates, CMTRF Funded Research, Drug Development

The CMT Research Foundation is proud to announce our latest project, a partnership between AcuraStem, a patient-based drug discovery platform company, and Dr. Alessandra Bolino, a renowned expert in CMT and the head of the Human Inherited Neuropathies Unit at the...

CMT Research Foundation To Host First Global Research Convention

Jun 15, 2021 | CMT Research Updates, CMTRF Funded Research, Drug Development, News, Press Releases, Research News

Several Hundred Charcot-Marie-Tooth* World Leaders Gather to Accelerate Collaboration and Advancements in Research; Pharma Partner DTx to Review Significant Progress in Drug Delivery Gene Therapy Innovator and Pioneer Dr. James M. Wilson to Keynote ATLANTA (June...

Promising Research Results Point to Potential Cures for Two Most Common Forms of CMT

Apr 19, 2021 | Drug Development, News, Research News

Momentum is building in the development of gene therapies for Charcot-Marie-Tooth disease. Landmark back-to-back research publications offer innovative approaches to potentially curing the two most common forms of CMT. Here is what the findings showed and what they mean for CMT patients and families.

Additional Funding Announced for Promising Gene Silencing Approach to Treat CMT1A

Feb 15, 2021 | CMT Research Updates, CMTRF Funded Research, Drug Development, Research News

After demonstrating tremendous progress, the CMT Research Foundation’s Scientific Advisory Board has decided to extend funding for a research project led by Dr. Kleopas Kleopa for six more months. If successful, this project could lead to the first genetic therapy for CMT1A. Read more.

CMT Research Foundation Launches Groundbreaking Research to Overcome Barriers to Delivering CMT Therapies to the Peripheral Nervous System

Feb 10, 2021 | CMT Research Updates, CMTRF Funded Research, Drug Development, Research News

The CMT Research Foundation has launched a groundbreaking new research collaboration today with James Dahlman, Ph.D., assistant professor in the Wallace H. Coulter Department of Biomedical Engineering at the Georgia Institute of Technology and Emory School of Medicine. This innovative partnership seeks to overcome the challenges in CMT drug delivery by using lipid nanoparticles (LNPs) to deliver therapies to the PNS that could slow, stop or even reverse the effects of CMT in patients.

What to Expect in CMT Research During 2021: 5 Questions with Chief Scientific Officer Keith Fargo

Jan 29, 2021 | Drug Development, News, Research News

What progress can patients and families expect to see in CMT research during 2021? What is the most promising research on the horizon? The CMT Research Foundation’s Chief Scientific Officer Keith Fargo, Ph.D., sat down with us to answer your most pressing questions about CMT research in the year ahead.

How to Measure Progress in CMT Research: 8 Signs Research is Working

Jan 11, 2021 | About CMT, CMT Research Updates, Drug Development, News, Research News

It costs more than $2.6 billion to develop an approved prescription medicine and typically takes between 10 to 15 years to get a drug to clinical trials. With no treatments or cures currently available for diseases like CMT, it’s easy to question how donations to support scientific research make a difference. These 8 signs let you know when research is working.

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...