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

Walking Manhattan to Find a Cure

From a young age, Rivka knew that there was something different about her compared to her three siblings; that difference was Charcot-Marie-Tooth (CMT) disease.Meet Rivka Ligier, a CMT1B patient who is preparing to participate in this year's Great Saunter, a one-day,...

Vanderbilt’s Charles Sanders Successfully Finds Molecules That Alter PMP22 Production or Cell Surface Trafficking; In Next Phase Will Test if They Can Improve CMT-like Problems in Schwann Cells

CMT1A is caused by a gene-copying event that results in the overproduction of the peripheral myelin protein 22 (PMP22) in Schwann cells. The excess PMP22 protein fails to traffic normally to the cell surface and instead collects inside the cells as clumps of proteins...

CMT Research Update/Armatus Bio

Armatus Bio has partnered with Andelyn Biosciences, Inc., a cell and gene therapy Contract Development and Manufacturing Organization to accelerate manufacturing of an Armatus gene therapy treatment for Charcot-Marie-Tooth Type 1A. The goal is to maximize program...


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