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.
CMT Research Foundation Targets CMT1X in Partnership with University of Illinois Chicago
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
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
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
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
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
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
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
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
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
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...
Important Research News Announcements
- Genomic Studies in Charcot-Marie-Tooth Disease July 31, 2024
- Charcot-Marie-tooth disease type 2A: An update on pathogenesis and therapeutic perspectives March 5, 2024
- Accelerate Clinical Trials in CMT (ACTCMT) Study March 1, 2024
- Creation of a Schwann Cell Gene Regulatory Network February 29, 2024
- Activation of XBP1s attenuates disease severity in models of proteotoxic Charcot-Marie-Tooth type 1B [Preprint] February 2, 2024
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