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.

Chief Science Officer comments on recent work

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

CMT Research Foundation Chief Science Officer Grace Pavlath, PhD comments on recent work by Morelli et al: Allele-specific RNA interference prevents neuropathy in Charcot-Marie-Tooth disease type 2D mouse models Some types of CMT result from the expression of a mutant...

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...

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,...

CMTRF Welcomes Dr. Grace Pavlath as Chief Science Officer

Jul 30, 2019 | 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...

Scenes from BIO2019

Jun 11, 2019 | Press Releases

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...

Partnerships and Priorities: The Pathway to CMT Results

May 31, 2019 | CMT Research Updates, Press Releases, Research News

From time to time the CMT Research Foundation will examine the impediments to finding a cure for CMT. As the Chairman of a nonprofit foundation whose ONLY mission is to fund research that will lead to a cure for Charcot-Marie-Tooth disease (CMT), I am often...

CMTRF investigates a gene therapy program for CMT1A

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

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...

The Inherited Neuropathy Consortium (INC) welcomes the CMT Research Foundation as a partner in their Patient Advocacy Groups

Jan 9, 2019 | CMT Research Updates, Press Releases

Today the Inherited Neuropathy Consortium (INC) named the CMT Research Foundation as a partner in their Patient Advocacy Groups. This gives the CMT Research Foundation access to INC resources, researchers and its clinical infrastructure through 21 CMT Centers of...