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.
‘Amazon Smile’ Program Discontinued
On February 20th, 2023, Amazon ended their Amazon Smile Charity Program. Thank you to the nearly 200 people who supported the CMT Research Foundation through this program. In total, the community was able to raise:Interested in finding a new way to make a difference,...
2022 at CMTRF
Dear Friends of CMTRF, In preparing for another busy year at CMTRF, I took some time recently to look back at the accomplishments of 2022. I am immensely proud of what our team has achieved (summarized below) and I believe it underscores how CMTRF adds significant...
How to Answer: “What is CMT”
By Kenneth Raymond As a patient and an advocate and a student of the condition, I am often asked, “What exactly is CMT?”. My response is usually along the lines of: “CMT stands for Charcot-Marie-Tooth disease, a rare inheritable neuromuscular peripheral neuropathy...
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...
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...
A New Trial for CMT – When Science Moves Quickly
By Keith Fargo, Ph.D., Chief Scientific Officer, CMT Research Foundation The pace of science can seem excruciatingly slow. It can take many years, or even decades, for new ideas to result in clinical trials. Every so often, though, the stars align and a new...
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....
Shift Pharmaceuticals Completes CMT Research Foundation-Funded Project, Will Continue Development of Gene Therapy for CMT1A
The CMT Research Foundation is pleased to announce that Shift Pharmaceuticals (Shift) has successfully completed their CMT Research Foundation-funded RNA-based therapy project. Shift created and tested a library of novel molecules designed to reduce the expression of...
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