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
Family Affected by CMT1A Makes a Cure Their ‘Endgame’
Published by CMT News (slightly edited for clarity) It was an unexpected $1 million gift, even for Kent and Michele Stahl. The Charcot-Marie-Tooth Research Foundation (CMTRF) was hosting a research convention in September when it announced Endgame, the...
Five Reasons Why We Think the End of CMT is in Sight
Nearly every instance of CMT is caused by the mutation of a single gene and most genetic causes of CMT have been identified. The era of genetic medicine has already begun. The FDA first approved genetic medicines in 2016/2017. This set the foundation for a precision...
Living with CMT2E
My name is Lily Sander and I have Charcot-Marie-Tooth type 2E. The road to my diagnosis wasn’t easy by any scale. I spent months as a small child in countless specialists' offices, anxiously waiting for an answer. At this time, I had severe clubfeet and could barely...
The Power of Scientific Collaboration – New Biomarkers for CMT
By Keith Fargo, Chief Scientific Officer At the CMT Research Foundation, we believe that more is achieved when people work together than when they work individually. This is especially true in biomedical research. Over the past few decades, a seismic shift has...
CMTRF Research Efforts to Benefit from a Portion of Ticket Sales from Alan Jackson’s Last Call: One More for the Road Tour
ATLANTA (March 11, 2022) CMT Research Foundation (CMTRF) the only nonprofit organization focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT)*, will see its efforts boosted by country music superstar Alan Jackson's fans this year as...
Shayla Hammock, living with CMT1A
I inherited CMT1A through my family, although I was unaware of what was affecting me until I was seven years old. As a baby, I was unable to lift my head at the same time as my peers and it took me a little longer to learn to walk. In fact, it seemed like every...
Gene therapy project for CMT2E has completed Milestone 1 successfully.
University of Missouri features CMT in a video about the project and the role of precision medicine to treat CMT. By: Susan Ruediger, Co Founder and Chief Mission Officer With the recent opening of their new NextGen Precision Health Institute, University of Missouri...
Living with Muscle Weakness is Not Rare to Me
Krista Allison, living with CMT4J I was born with Charcot-Marie-Tooth disease (CMT) and my symptoms started to appear about 12-months-old when I began to learn to walk. Since the age of 15, I have had roughly 12 foot surgeries to correct foot abnormalities, although I...
Maximiliano Barrientos Joins CMT Research Foundation
The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease* is pleased to announce that Maximiliano Barrientos has joined its Board of Directors. Mr. Barrientos is a Co-founder, Investor and CFO...
AcuraStem Completes CMT Research Foundation-Funded Project, Finds Eleven Potential Therapeutics for CMT2A
By Keith Fargo, Chief Scientific Officer The CMT Research Foundation is pleased to announce that the research team at AcuraStem has completed their CMT Research Foundation-funded drug screening project and has found eleven potential therapeutics for CMT2A....
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