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.
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.
More Than Hope: An Action Plan to End CMT
As we approach the end of 2020 and look ahead to a new year, it’s natural to feel hopeful about what the future may hold — hope for an end to the global pandemic; hope for happiness and good health for our families; and hope for an end to Charcot-Marie-Tooth disease (CMT). The CMT Research Foundation shares these hopes, but we know delivering treatments and cures for CMT quickly requires more than hope. It requires vision, commitment, wisdom, a strong plan, and most importantly — action.
See a preview of the CMT Research Foundation’s 2021 action plan.
‘Tis the Season for Scientific Breakthroughs
During a year when so many things have been postponed, changed or cancelled, there is one thing that hasn’t stopped — the CMT Research Foundation’s efforts to end CMT. Research continues, even during the holidays, to find treatments and cures as quickly as possible. These researchers will be working this holiday season to uncover scientific breakthroughs. Here’s how several of our researchers will be celebrating by continuing their work for you.
20 Most Popular CMT Stories & Resources from 2020
2020 has been a year of information overload. To help the Charcot-Marie-Tooth (CMT) community navigate these staggering statistics, we’ve developed a round-up of the 20 most read articles and stories about CMT on cmtrf.org during 2020. You were most interested in three key areas: 1) stories from the CMT community, 2) the latest in research development and discovery and 3) understanding CMT and specifics about the disease.
Thank You for Giving Me More Than Hope
This year has brought great momentum in the fight against CMT. At the CMT Research Foundation, we are hearing from scientists that it feels like something special is in the air. Conferences and journals are full of new discoveries, fundamental problems in CMT are being solved, new genes are being discovered, and new drugs are being developed. Read more about the progress.
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...
DTx Pharma Shows Continued Progress in Reducing PMP22 Levels in Animal Models of CMT1A
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
“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
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.
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