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.

New Evidence for Gene Replacement in a Mouse Model of Charcot-Marie-Tooth Disease

May 24, 2019 | CMT Research Updates, Press Releases, Research News

In an article published yesterday in Neurology Today, Dr. Kleopa demonstrated that gene replacement could be a viable treatment for recessive forms of CMT. CMT type 4C is a demyelinating disease, and this treatment both decreased the number of unmyelinated fibers and...

CMTRF Responds to FDA Fast Track Designation of PXT3003

Feb 11, 2019 | CMT Research Updates, News

The FDA Grants Fast Track Designation to Pharnext's PXT3003 for CMT1A Pharnext, an advanced clinical-stage biopharmaceutical company, created a drug combination of three drugs: baclofen, naltrexone and sorbitol known as PXT3003. Baclofen is known to relieve muscle...

CMTRF investigates a gene therapy program for CMT1A

Jan 17, 2019 | CMT Research Updates, CMTRF Funded Research, Press Releases

CMT Research Foundation Partners with Cyprus Institute for Neurology and Genetics’ Kleopas Kleopa to Launch Study of Possible Gene Therapy for CMT1A This is the second major investment by CMTRF. ATLANTA (January 16, 2019) The newly launched CMT Research Foundation...

CMTRF Inks Milestone-Driven Partnership with AcuraStem to Repurpose Established Drugs for the Treatment of CMT

Dec 20, 2018 | CMTRF Funded Research, Press Releases

Focus will be on expansion of AcuraStem’s patient-based discovery platform, iNeuroRx™, to leverage patient neurons and advanced informatics technology to identify promising drugs ATLANTA (December 20, 2018) The newly launched CMT Research Foundation (CMTRF), 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...