Research in pediatric neurosurgery and brain tumors has highlighted several critical aspects of diagnosis and treatment. One significant study focused on Ritscher-Schinzel syndrome (RSS), a congenital malformation syndrome that can present with various neurological symptoms, including cerebellar malformations. The genetic underpinnings of RSS remain partially understood, with emerging evidence suggesting causative mutations in the Commander multiprotein assembly (ref: Kato doi.org/10.1126/scitranslmed.adt2426/). In the realm of gliomas, particularly high-grade gliomas, Zhou et al. demonstrated that UBE2T-mediated ubiquitination enhances nucleolar function, promoting the progression of IDH1/TP53-mutant gliomas. This study posits that targeting UBE2T could be a promising therapeutic strategy for these aggressive tumors (ref: Zhou doi.org/10.1158/1078-0432.CCR-25-0261/). Furthermore, innovative treatment approaches such as the use of carbonic anhydrase inhibitors have been explored, with Richman et al. showing that acetazolamide can sensitize Group 3 medulloblastoma to radiotherapy, potentially improving survival rates in pediatric patients (ref: Richman doi.org/10.1158/0008-5472.CAN-24-3894/). The role of simvastatin in suppressing spinal cord metastasis of medulloblastoma has also been investigated, revealing its potential efficacy at clinically significant doses (ref: Comer doi.org/10.1038/s41419-025-07829-0/). Collectively, these studies underscore the complexity of pediatric brain tumors and the need for targeted therapies to improve outcomes.