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.
Two papers were recently published regarding CMT1B and Axonal Neuregulin 1 Type III (Nrg1TIII), a protein which builds myelin (the insulation) on the nerves.
In summary, there is a molecule which blocks the Nrg1TIII in CMT1B, causing the demyelination of the nerves. Both papers found that if the strength or amount of Nrg1TIII can be controlled, the nerves affected by CMT may be restored. When more of the protein is added...
Why FDA acceptance of gene therapy in SMA matters to CMT
Novartis's announcement about FDA filing acceptance could translate to CMT On Monday, Novartis, the parent company of AveXis, announced that a license application for their gene therapy approach to treat Spinal Muscle Atrophy (SMA) was accepted by the FDA. This is a...
Fontan Surgery for Heart Defects May Not Be Advisable for CMT1A Patients, Case Study Finds
Charcot-Marie-Tooth News reports the findings of a study where patients with Charcot-Marie-Tooth disease (CMT) who undergo Fontan surgery to correct a heart defect in childhood may later develop difficulties with the diaphragm — the muscle that allows us to breath —...
Where We Started, How Far We Have Come and Where We are Going
The CMT Research Foundation offers hope to those living with CMT that a treatment and cure are on their way!
$1,000,000 pledge to CMT research
CMTRF Receives $1 Million Research Grant from Grandview Steers Foundation The pace of research for CMT just got faster. Atlanta (November 8, 2018) The newly launched CMT Research Foundation (CMTRF), the only 501(c)(3) not-for-profit organization focused solely on...
Key CMT Researchers added to Scientific Advisory Board
Gene Therapy, Adult Stem Cell and Clinical Experts are added to the CMT Research Foundation’s Scientific Advisory Board
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
Address
4062 Peachtree Road
Suite A209
Atlanta, GA 30319
Phone Number
404.806.7180
Media Inquiries
© 2024 CMT Research Foundation | Privacy Policy