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
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 stage...
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. With the recent opening of their new NextGen Precision Health Institute, University of Missouri is quickly rising as a leader in precision medicine....
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
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. AcuraStem developed a platform to screen compounds...
Welcoming New Expert Advisors
By Keith Fargo, Chief Scientific Officer The CMT Research Foundation’s Scientific Advisory Board is composed of distinguished scientists and clinicians who provide independent expert perspectives as key volunteers. Their contributions take several forms. Most visibly,...
CMT Research Foundation Elects Board Member Cleary Simpson to Chief Executive Officer
By Patrick Livney, Board Chair and Co Founder As we move from being a startup to the leading foundation seeking to cure CMT, we can look back with great pride on our progress to date: We have raised almost $10 million in three years, funded 12 projects (with two in...
Michele and Kent Stahl and Family Give $1,000,000 to the CMT Research Foundation to Develop Treatments for CMT1A
Atlanta (January 4, 2021) Michele and Kent Stahl and their family today announce a $1 million donation to support the CMT Research Foundation’s $10 million ENDGAME: the Campaign to End CMT1A. Since the soft launch of ENDGAME in September, families and friends with...
Why Michele and Kent Stahl and their family made a $1,000,000 gift to CMT Research Foundation to develop treatments for CMT1A
Michele and Kent’s son, Austin, was diagnosed with CMT1A when he was 16 years old. Having difficulty with his balance and walking, it took years for him and his family to understand the cause. His mother Michele recounts, “we spent seven years searching for an answer....
2021 Year in Review
2021 has been a banner year for progress in CMT drug development, much of which has been possible because of your partnership with the CMT Research Foundation – thank you! Despite the challenges presented by a global pandemic, we can be proud of all that we were able...