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.
Mother and Three Sons, All Diagnosed with CMT2N within a Year
Just one year ago, Tara’s oldest son, Luca, started coming home from school crying in pain. He would rush to take off his shoes and socks, saying that his feet were “buzzing”. At five years old, Luca’s foot had high arches and a deformity, causing his bones to...
DTx Pharma
We are pleased share this story that published on May 3: https://patientworthy.com/2023/05/03/dtx-pharma-developing-cmt1a-rna-therapeutics/ One of the early programs CMTRF funded was DTx, a young company that had developed a new platform for RNA therapeutics. DTx was...
The CMT Research Foundation (CMTRF) Joins the European CMT Federation (ECMTF)
The CMTRF is happy to announce that they are joining the ECMTF, a non-profit organization grouping European and worldwide national associations supporting people affected by the Charcot-Marie-Tooth (CMT) disease or neuromuscular disorders. One of the primary goals of...
CMT Research Foundation Collaborates with Augustine Therapeutics on New Potential Therapy for Charcot-Marie-Tooth Disease
ATLANTA (March 2, 2023) The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT)*, is pleased to announce a collaboration with Augustine Therapeutics, a Belgian pharmaceutical company...
A Rare Disease Throughout Time
More than 3 million people around the world have Charcot-Marie-Tooth (CMT) disease, but even with its quirky name, CMT is still relatively unknown. A progressive, degenerative disease involving the peripheral nerves that branch out from the brain and spinal cord to...
‘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...
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...
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