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.
Muscular Dystrophy Association and CMT Research Foundation Announce Joint Research Study Grant
(New York) December 14, 2022 The Muscular Dystrophy Association (MDA) and CMT Research Foundation (CMTRF) announce joint funding for a study by Alessandra Bolino, PhD, at San Raffaele Hospital (Ospedale San Raffaele), Italy, on restoring membrane trafficking in...
Why I Fundraise
My name is Anna and I have CMT1A. I fight for CMT to raise awareness to help others. I fight for CMT to inspire those living with CMT to feel less daunted about the future I fight for CMT to support research for treatments. I fight for CMT to help find a cure. I am...
CMT Research Foundation partners with Samsara Therapeutics to develop a novel class of drugs in CMT1A
The CMT Research Foundation is pleased to announce a new project with Samsara Therapeutics, a US/UK-based biotech company that is developing a novel class of drugs for CMT1A. CMT1A is caused by duplication of a stretch of DNA that includes the PMP22 gene, and people...
CMT Research Foundation Awards Grant to Nationwide Children’s Hospital’s Dr. Afrooz Rashnonejad to Develop Gene Therapy to Cure or Alleviate CMT1B
ATLANTA (July 18, 2022) The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease*, has awarded a grant of over $500,000 to find a single gene therapy for every form of CMT1B. The award was made...
Christopher Austin to Keynote CMT Research Foundation’s 2022 Global CMT Research Convention
Christopher Austin, Founding Director of the National Center for Advancing Translational Sciences, to Keynote CMT Research Foundation’s 2022 Global CMT Research Convention ATLANTA (July 12, 2022) Christopher Austin, M.D., CEO of Vesalius Therapeutics, CEO-Partner of...
Project Testing PIKfyve Inhibitor in CMT4B1 Clears First Hurdles
CMTRF is happy to report that a CMTRF-funded project designed to test a PIKfyve inhibitor in CMT4B1, a particularly devastating form of the disease, has passed the first stage and is moving on to the next stage, a preclinical trial in CMT4B1 model mice. Before the...
Legacy – an Anthem for Patients with CMT
Recording-artist/songwriter/producer Dina Fanai AKA AVALONA, released Legacy today across all platforms Legacy was written as an anthem for patients with Charcot-Marie-Tooth disease (CMT), one of the world’s most common inherited neurological diseases. CMT causes...
CMT Mothers
The CMT Research Foundation knows that moms in the world of CMT have unique challenges and trials. And we salute their tenacity, patience, and unyielding dedication. Here are the stories of two CMT moms; one whose daughter lives with CMT and another who is a patient....
Family Affected by CMT1A Makes a Cure Their ‘Endgame’
Published by CMT News (slightly edited for clarity) It was an unexpected $1 million gift even for Kent and Michele Stahl. The Charcot-Marie-Tooth Research Foundation (CMTRF) was hosting a research convention in September when it announced Endgame, the organization’s...
Five Reasons Why We Think the End of CMT is in Sight
Nearly every instance of CMT is caused by the mutation of a single gene and most genetic causes of CMT have been identified. The era of genetic medicine has already begun. The FDA first approved genetic medicines in 2016/2017. This set the foundation for a precision...
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