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Abstract
The clinical hallmark of neuroblastoma is heterogeneity, with the likelihood of cure
varying widely according to age at diagnosis, extent of disease, and tumour biology.
A subset of tumours will undergo spontaneous regression while others show relentless
progression. Around half of all cases are currently classified as high-risk for disease
relapse, with overall survival rates less than 40% despite intensive multimodal therapy.
This Seminar focuses on recent advances in our understanding of the biology of this
complex paediatric solid tumour. We outline plans for the development of a uniform
International Neuroblastoma Risk Group (INRG) classification system, and summarise
strategies for risk-based therapies. We also update readers on new discoveries related
to the underlying molecular pathogenesis of this tumour, with special emphasis on
advances that are translatable to the clinic. Finally, we discuss new approaches to
treatment, including recently discovered molecular targets that might provide more
effective treatment strategies with the potential for less toxicity.