News & Stories

See the latest news about CMT drug development and read stories from the CMT community that highlight why we must deliver treatments and cures during our lifetime.

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

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...

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CMT Research Update/Armatus Bio

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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Updates on CMT Research

Updates on CMT Research

There have been a number of interesting developments in CMT research in the past few weeks. Here are three: Update 1: Pharnext recently announced topline results of its pivotal phase 3 (PREMIER trial) of PXT3003, its drug candidate for Charcot-Marie-Tooth 1A (CMT1A)...

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