CMT News & Updates
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.
Filter by Category
Drug Screening in a Dish
At the CMT Research Foundation we are very much interested in using patient-derived stem cells called iPSCs for drug screening in order to develop therapies for CMT. Indeed, one of…
Gene Editing Used for the First Time in CMT
Gene editing as a therapeutic tool for treating disease has been very much in the news recently. The most commonly used gene editing tool to permanently fix the underlying genetic…
Chief Science Officer comments on recent work
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…
Emerging Landscape of Modifier Genes in CMT
In CMT, there is a large variation in disease onset, severity, or progression, often seen among individuals with the same genetic subtype of a disease, even within a single family. …
Inherited Neuropathy Consortium Awarded $7.2M to Continue Studying, Improve Care for CMT
The National Institutes of Health has awarded the Inherited Neuropathies Consortium (INC) $7.2M over the next five years. This grant will be focused on improving clinical care and clinical research…
Read More : Inherited Neuropathy Consortium Awarded $7.2M to Continue Studying, Improve Care for CMT
Where is the Progress? The Cure For CMT Calls For Collaboration
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…
Read More : Where is the Progress? The Cure For CMT Calls For Collaboration
AcuraStem Achieves First Milestone
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…
Roadblocks on the Road to Curing Charcot-Marie-Tooth (CMT)
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…
Read More : Roadblocks on the Road to Curing Charcot-Marie-Tooth (CMT)
CMTRF’s Dr. Grace Pavlath on new results in gene therapy research for CMT1X
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…
Read More : CMTRF’s Dr. Grace Pavlath on new results in gene therapy research for CMT1X
CMT1A Research Update
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.…