Retinoblastoma.

This Lancet Seminar reviews retinoblastoma, the most common childhood intraocular malignancy, asking how advances in its biology, diagnosis, and treatment can be used to close the large global gaps in burden and survival. The authors summarize key findings: the disease is mainly driven by biallelic RB1 inactivation (with MYCN amplification in about half of the rare RB1-negative cases), aqueous humour liquid biopsy shows promise for minimally invasive diagnosis and monitoring, and eye-preserving intra-arterial, intravitreal, and intracameral chemotherapy are effective and safe. They conclude that mortality remains highest in low sociodemographic index regions, underscoring the need for equity-focused efforts to ensure timely diagnosis and access to high-quality care worldwide.