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.

Dec 14, 2018 | CMT Research Updates

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

Dec 7, 2018 | CMT Research Updates

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

Nov 19, 2018 | CMT Research Updates, Research News

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

Nov 15, 2018 | Press Releases, Research News

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

Nov 8, 2018 | Press Releases, Research News

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...