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.

Where is the Progress? The Cure For CMT Calls For Collaboration

Oct 11, 2019 | CMT Research Updates, Press Releases, Research News

More collaboration within the CMT research and patient community would help us get to treatments and a cure much faster. Thomas Durcan, an Assistant Professor for neurodegenerative disorders at McGill University recently wrote a very interesting appeal for...

AcuraStem Achieves First Milestone

Sep 18, 2019 | CMT Research Updates, Press Releases, Research News

Just nine months ago, we announced a milestone-based, research collaboration with AcuraStem to test thousands of compounds aimed at producing effective treatments for CMT2A. We are very pleased to report that AcuraStem has completed phase one of the CMT2A project. To...

Roadblocks on the Road to Curing Charcot-Marie-Tooth (CMT)

Sep 11, 2019 | CMT Research Updates, Press Releases, Research News

It seems like every other day we hear about a new drug that has been approved for a rare neurologic disease. Increased understanding of the genetics underlying these diseases has led to numerous new gene therapies. Why have we not yet seen similar successes in CMT? In...

CMTRF’s Dr. Grace Pavlath on new results in gene therapy research for CMT1X

Sep 8, 2019 | CMT Research Updates, Research News

There’s a lot of excitement around gene-targeted therapies for their ability to treat the underlying root cause of various diseases and result in life altering outcomes. The neuromuscular disease field has recently seen the FDA approval of gene-targeted therapies for...

CMT1A Research Update

Sep 2, 2019 | CMT Research Updates, Press Releases, Research News

CMT1A is caused by a duplication of the PMP22 gene leading to overexpression of the PMP22 protein and failure of the Schwann cells to produce myelin (the insulation of the nerve) well. Without proper myelination, the nerves don’t send signals quickly or strongly,...

CMT1B project is funded & ready for take off!

Aug 2, 2019 | CMT Research Updates, Press Releases, Research News

Paving pathways for mission critical projects CMT Research Foundation is partnering with Dr. Maurizio D’Antonio of I.R.C.C.S. Ospedale San Raffaele, in Milan, Italy to fund the lab’s research of Myelin Protein Zero (MPZ), the gene associated with CMT1B. Dr....

CMTRF Welcomes Dr. Grace Pavlath as Chief Science Officer

Jul 30, 2019 | CMT Research Updates, Press Releases

CMT Research Foundation (CMTRF), a not-for-profit organization focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease, has added Grace Pavlath, Ph.D. to the team as the new Chief Scientific Officer. CMT is a progressive genetic nerve disease...

New research efforts by Neurogene, give hope to patients suffering with an ultra-rare type of CMT: CMT4J

Jul 19, 2019 | CMT Research Updates, Press Releases, Research News

Neurogene, a leading company developing genetic medicines for people with rare, devastating neurological diseases, announced today that it is recruiting patients for a natural history study for CMT4J. "Key data now being collected to support endpoint assessment for...

Is “Disease-In-A-Dish” in Combination with Animal Models A Faster Track to a Cure for CMT?

Jul 1, 2019 | CMT Research Updates, Press Releases, Research News

From time to time the leadership of CMTRF will explore the challenges of finding treatments and a cure for CMT. To the extent we are able, we will try to overcome these obstacles to accelerate successful CMT research. Treatment of diseases, especially rare diseases...

Scenes from BIO2019

Jun 11, 2019 | News

Last week Paul August and I attended the BIO International Convention, a conference with over 16,000 leaders from the biotech and pharmaceutical industry. We met a number of companies interested in advancing drug development for CMT. There are four primary factors...