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.
There is No Guidebook on How to Parent a Child with CMT
By Thomas Sander When our daughter, Lily, was four-years-old she was unusually clumsy, something we initially wrote off as developmentally appropriate. Soon, however, her feet turned in dramatically, leaving her mobility severely restricted. My wife and I spent an...
Taking on the Yorkshire Three Peaks Challenge for Charcot-Marie-Tooth (CMT)
Two women in the UK, Lucy Brown and Chrysi Albustin, will be tackling the Yorkshire Three Peaks Challenge to raise awareness and funds for Charcot-Marie-Tooth (CMT). Lucy’s mother was diagnosed with CMT1A in 2002; before Lucy was born. CMT is a progressive,...
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...
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...
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 discovery...
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...