Severe junctional epidermolysis bullosa (sJEB) is a rare genetic, often post-partum lethal epithelial disease that is predominantly caused by biallelic non-sense/premature termination codon (PTC) sequence variants in the LAMB3 gene [1]. The devastating clinical manifestations include generalized blistering at birth, periorificial erosions around the mouth, eyes, and nares, significant loss of mucosal surfaces of the inner organs, a hoarse cry, cough and respiratory difficulties. Sepsis and a general failure to thrive result in patient mortality in early infancy [2]. Targeted ribosome editing in yeast and human cells identified artesunate as an efficient enhancer of lamβ3 protein translation [3–6]. In this work, Sinz et al. [7] showed that an application of artesunate to both sJEB-LAMB3 HaCat model cells and to immortalized sJEB-LAMB3 patient keratinocytes revealed a significant increase of lamβ3 protein (Fig.). Based on these data, artesunate was used in a newborn patient with sJEB, homozygous for a LAMB3 PTC mutation in an off-label indication. Artesunate was well tolerated. The clinical response was mixed: During the first five months of treatment, no effect on wound healing (nates and flanks) was observed according to the treating physician’s assessment. An investigation of patient skin with immunofluorescence was performed after five months of treatment, revealing a marginal increase in the expression of lamβ3 protein. As oral dosing reached 40 mg daily, the patient’s wounds on the nates began to heal gradually. However, the patient‘s general condition worsened, and she could not be saved. To promote the effects of artesunate in vivo that have been detected in vitro for the clinical benefit of sJEB-LAMB3 patients, higher dosing will be necessary. To this end, artesunate derivatives are being currently developed.