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.
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...
ARM-101: A Potential “Precision Drug Therapy” For CMT1A
The CMT Research Foundation has invested in a project at Armatus Bio, Inc. that will advance a novel miRNA gene therapy candidate for CMT type 1A. Principal Investigator Dr. Brian Price and his colleagues aim to harness the potential of microRNAs (miRNAs), snippets of...
CMT Research Foundation Launches Research Advisory Committee of Renowned Scientific Experts
ATLANTA (October 18, 2023) The CMT Research Foundation (CMTRF), a patient-led, non-profit focused on delivering treatments and cures for Charcot-Marie-Tooth (CMT) disease*, today announced the formation of a Research Advisory Committee (RAC) consisting of: Dr. William...
Oryzon Develops Novel HDAC6 Inhibitor ORY-4001 Showing Therapeutic Potential for CMT in Mouse Model
Scientists have been studying a potential treatment option for CMT by inhibiting (blocking) a protein called histone deacetylase 6 (HDAC6). They have found that HDAC6 plays an important role in regulating the pathways involved in breaking down proteins and...
DTx Pharma Acquired by Novartis for CMT1A Therapy
Novartis acquires DTx’s FALCON technology to advance treatment development for CMT1A
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...
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...
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...
Important Research News Announcements
- Genomic Studies in Charcot-Marie-Tooth Disease July 31, 2024
- Charcot-Marie-tooth disease type 2A: An update on pathogenesis and therapeutic perspectives March 5, 2024
- Accelerate Clinical Trials in CMT (ACTCMT) Study March 1, 2024
- Creation of a Schwann Cell Gene Regulatory Network February 29, 2024
- Activation of XBP1s attenuates disease severity in models of proteotoxic Charcot-Marie-Tooth type 1B [Preprint] February 2, 2024
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