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
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William Skach, MD, Joins CMT Research Foundation Board of Directors
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CMT Research Foundation to Benefit from a Portion of Ticket Sales from Alan Jackson’s Last Call: One More for the Road – The “Finale”
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CMT Research Foundation Funds Elpida Therapeutics to Launch Manufacturing of a Gene Therapy Drug for CMT4J
CMT Research Foundation announced they have funded Elpida Therapeutics to launch manufacturing of a gene therapy drug for Charcot-Marie-Tooth disease type 4J. The announcement came at the 2025 Global CMT Research Convention. “Our ELP-02 gene therapy is trial-ready,”...
Dr. Meghan Drummond Joins the CMT Research Foundation as Vice President of Research and Drug Development
Dr. Meghan Drummond, PhD, has joined the CMT Research Foundation as vice president of research and drug development. She will lead the Foundation’s research and drug development strategy, overseeing the advancement of promising therapeutic approaches for...
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Applied Therapeutics to Meet With FDA on New Drug Application for the Treatment of CMT-SORD
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Dr. Charles Abrams Discovers That Inosine Treatment Leads to Benefits in a CMT1X Animal Model
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Dr. Yulia Grishchuk Joins CMTRF’s Scientific Advisory Board
CMT Research Foundation is pleased to announce that Dr. Yulia Grishchuk, Ph.D., has joined the Foundation’s Scientific Advisory Board. The SAB is composed of distinguished scientific and clinical experts specializing in Charcot-Marie-Tooth disease and drug...
