Small Molecule Therapies for CMT
Small molecule therapies typically are developed from simple building blocks or are derived from natural sources. With their small size, they are easily able to pass through the cell membrane and access targets inside the cell.
Small molecule drugs are designed to modify a disease process via regulation of a biological target such as an enzyme, channel or receptor. They make up the majority of today’s drug treatments and may be used alone or as individual elements in combination therapies.
CMT Research Foundation is leading the charge to ensure safe and effective therapies for all forms of CMT make it to the market to help those living with CMT today. We are working every day to support the families, the foundations, the academics, the scientists, the biotechs and the pharma companies who share our goal and who are working to make it happen.
Related News & Research
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...
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...