Changing One Family’s Legacy

As with many families, I am but one leaf in a family tree colored by CMT1A across its many limbs and branches. I have watched successive generations struggle with the disease’s progressively debilitating physical symptoms and persistent emotional toll: grandparents,...

DTx Pharma Milestone 1 Completed

Our partners at DTx Pharma continue to make great progress on suppressing the overexpression of PMP22, which causes CMT1A. DTx Pharma’s proprietary technology has the potential to render RNA-based therapeutics such as ASO and siRNA more potent, safer and longer-acting...