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.
Progress is Possible Because of You
As we begin 2021, I am overwhelmed with gratitude for your continued commitment to the CMT Research Foundation community. Despite a challenging last year, friends like you rose to the challenge and have made it possible for the CMT Research Foundation to fund new and...
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.
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.
CMT Research Foundation Welcomes Dr. James Hendrix to Scientific Advisory Board
The CMT Research Foundation is pleased to announce that James Hendrix, Ph.D., has joined our Scientific Advisory Board to help guide research funding to deliver treatments and cures for CMT.
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...
How We Get from Today to Approved CMT Treatments: An Interview with FDA Director Dr. Peter Marks
The CMT Research Foundation is asking and answering the most pressing questions patients have about the need for treatments and cures. In episode 3, CMT Research Foundation CEO Susan Ruediger interviews the FDA’s Dr. Peter Marks to discuss gene therapies as a potential treatment option for CMT, what the approval process looks like and how patients can expedite it.