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

The CMT Research Foundation’s 2024 Global CMT Research Convention to Gather Renowned Scientific Experts and Patients to Discuss Status of Treatments and Cures for Charcot-Marie-Tooth (CMT) Disease

The CMT Research Foundation recently announced most of the speakers at its upcoming 2024 Global CMT Research Convention to be held September 26-28, in Cambridge, MA. On Thursday, September 26, CMTRF will host a Young Researcher Innovation Forum for CMT Research....

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