PMP22 and Charcot-Marie-Tooth disease: The challenges in developing an effective treatment for CMT can be attributed to three main factors: (1) extensive genetic heterogeneity, with more than 1500 identified underlying point alterations, counting the 1.4 Mb CMT1A duplication, coupled with overlapping disease phenotypes; (2) the relative scarcity of individuals per genotype, which reduces interest from both research communities and pharmaceutical companies; and (3) the complexity involved in transitioning preclinical research from rodent and cellular models to human clinical investigations [5].