Candidate treatments to test include drugs previously shown to work in other CMT subtypes,47,48 other MTMR-related neuromuscular disorders (e.g. valproic acid,43 tamoxifen49 and wortmannin50 in X-linked myotubular myopathy due to MTM1 mutation), and drug libraries targeting MTMR5-related pathways, for example, epigenetics (this study), endocytosis and autophagy.51 This evidence concerns the gene MTM1 and X-linked myotubular myopathy.