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.
Is there a connection between CMT1X and Multiple Sclerosis?
A recent study by Georgios Koutis et al at the National and Kapodistrian University of Athens recently published a paper in The Journal of Neurology, Neurosurgery and Psychiatry suggesting emerging evidence of a connection between CMTX1 and Multiple Sclerosis (MS)....
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 —...
Important Research News Announcements
- The Role of ESCRTs in Regulating Nervous System Function November 30, 2024
- 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
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