To further validate ATP13A3 as a therapeutic target and assess its biomarker value in predicting responsiveness to DFMO in neuroblastoma, in vivo xenograft studies will be required to determine the efficacy of DFMO, AMXT 1501 and their combination in a panel of patient‐derived xenograft models with varying expression levels of ATP13A3 and/or ODC1. This evidence concerns the gene ATP13A3 and neuroblastoma.