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Treatment entails surgery, intensive chemotherapy and radiotherapy, associated with substantial short- and long-term toxicities. There is an unmet need to develop targeted therapies for RT to improve patient outcomes and mitigate the toxicities of current therapy. Detailed research followed by a workshop had the objective of enabling the development of targeted therapeutics for RT. Given the inherent commonality of their biology (i.e. biallelic inactivation of\n                    <jats:italic>SMARCB1<\/jats:italic>\n                    or more rarely\n                    <jats:italic>SMARCA4<\/jats:italic>\n                    ) the therapeutic approach should be similar for intra-cranial and extra-cranial tumours. DDB1\u2013CUL4-associated factor 5 is a promising target, and the development of small molecule binders\/degraders is a priority. Enhancer of zeste 2 polycomb repressive complex 2 subunit (EZH2) degraders may have greater therapeutic potential than inhibitors. Fibroblast growth factor receptor and platelet-derived growth factor receptor inhibitors may have value in subgroups. Mouse double minute 2 homologue (MDM2) is a priority target for novel therapeutic development and combination trials. Combinations of EZH2, MDM2 inhibitors and selective inhibitors of nuclear export should be evaluated robustly preclinically and drive early clinical studies.\n                  <\/jats:p>","DOI":"10.1038\/s41416-026-03348-7","type":"journal-article","created":{"date-parts":[[2026,2,12]],"date-time":"2026-02-12T17:47:50Z","timestamp":1770918470000},"page":"1510-1528","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["Paediatric Therapeutic Development Workshop on rhabdoid tumours"],"prefix":"10.1038","volume":"134","author":[{"given":"Claudia","family":"Montiel Equihua","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jan 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