An immune modulatory off-label approach for treating CMT1X
The Martini laboratory has also initiated a program of evaluating drugs approved for other diseases for their possible use in treating CMT. The principle being followed is that drugs with demonstrated positive effects in related diseases can be rapidly re-positioned to treatment of CMT disorders following preclinical studies in rodent models of CMT. This approach aims at leveraging scientific knowledge of CMT disease pathology to identify potentially useful drugs that are already available, and perform enabling animal studies that can accelerate their application in the treatment of CMT patients.