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.
A Robust Therapeutic Pipeline for CMT 1A
The majority of people with CMT have the 1A subtype which is due to duplication of the peripheral myelin protein 22 (PMP22) gene leading to breakdown of the myelin that surrounds peripheral nerves. With time this loss of myelin causes nerve dysfunction and...
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 the first projects we funded was such a drug screening effort for CMT2A at Acurastem....
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 cause of a disease is CRISPR/Cas9. A recent paper demonstrates the first...
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 types of CMT result from the expression of a mutant...
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. Genetic modifiers play important roles in such variations, known as heterogeneity....
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 for CMT. The CMT Research Foundation is a proud partner of the INC....
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 University recently wrote a very interesting appeal for...
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 has completed phase one of the CMT2A project. To...
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 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
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...
Important Research News Announcements
- Genomic Studies in Charcot-Marie-Tooth Disease July 31, 2024
- Charcot-Marie-tooth disease type 2A: An update on pathogenesis and therapeutic perspectives March 5, 2024
- Accelerate Clinical Trials in CMT (ACTCMT) Study March 1, 2024
- Creation of a Schwann Cell Gene Regulatory Network February 29, 2024
- Activation of XBP1s attenuates disease severity in models of proteotoxic Charcot-Marie-Tooth type 1B [Preprint] February 2, 2024
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