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.
Partnership with DTx Pharma to optimize antisense oligonucleotides as a gene therapy for CMT1A
ATLANTA (December 20, 2019) The CMT Research Foundation (CMTRF), a nonprofit focused solely on delivering treatments and cures for Charcot-Marie-Tooth, today announced it has partnered with DTx Pharma, an RNA medicines company breaking open new therapeutic areas for...
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...
Because of You
This year I am fortunate to celebrate the holidays with my family. As we prepared to gather three generations for Thanksgiving, my sister and I made an unusual stop: we rented a ramp so my mother could get into my sister’s home. My mom's CMT has progressed enough...
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...
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...
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. Without proper myelination, the nerves don’t send signals quickly or strongly,...
Important Research News Announcements
- The Role of ESCRTs in Regulating Nervous System Function November 30, 2024
- 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
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