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.
The Importance of Fundraising
By: Miron Hall Because CMT is a rare disease, it often receives less attention and funding than more common medical conditions. Without adequate resources, researchers struggle to develop effective treatments and ultimately find a cure. This is where fundraising plays...
Update from the Novartis CMT1A Development Team
As you know, the CMT Research Foundation is proud to have introduced DTx leadership to Charcot-Marie-Tooth disease in 2019. We funded their first work in CMT to demonstrate the FALCON platform's potential to deliver a drug to the Schwann cells. And from there, the...
Joao Cardoso, Paralympic Table Tennis Player with CMT2A
Meet Joao Cardoso, a paralympic table tennis player from Portugal, living in France, and competitor in this year’s International Para Championship. In his 5 years of competing, Joao has earned many awards and titles, including the Medal of Sporting Merit and this...
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...
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...
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...