To date, no effective treatment is available for PMD, although deferiprone, an iron chelator, reduced oligodendrocyte apoptosis and facilitated myelin formation in the jimpy mouse model of connatal PMD (Nobuta et al., 2019), and ketogenic diet (Stumpf et al., 2019), cholesterol supplementation (Saher et al., 2012) and transcriptional suppression by artificial microRNA (Li et al., 2019) have proven beneficial in Plp1-overexpressing animal models. The gene discussed is PLP1; the disease is Pelizeaus-Merzbacher spectrum disorder.