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Children 2018, 5(10), 142;

Molecularly Targeted Therapy for Neuroblastoma

The University of Minnesota Masonic Children’s Hospital/Masonic Cancer Center, Riverside Avenue, Minneapolis, MN 55454, USA
Received: 4 August 2018 / Revised: 26 September 2018 / Accepted: 27 September 2018 / Published: 15 October 2018
(This article belongs to the Special Issue Recent Advances in Diagnosis and Treatment of Neuroblastoma)
PDF [229 KB, uploaded 15 October 2018]


Neuroblastoma is the most common extra-cranial solid tumor encountered in childhood and accounts for 15% of pediatric cancer-related deaths. Although there has been significant improvement in the outcomes for patients with high-risk disease, the therapy needed to achieve a cure is quite toxic and for those that do experience a disease recurrence, the prognosis is very dismal. Given this, there is a tremendous need for novel therapies for children with high-risk neuroblastoma and the molecular discoveries over recent years provide hope for developing new, less toxic, and potentially more efficacious treatments. Here I discuss many of the molecular aberrations identified thus far in neuroblastoma, as well as the agents in development to target these changes. The progress made in both the preclinical arena and in early phase drug development provide much promise for the future of precision medicine in neuroblastoma. View Full-Text
Keywords: neuroblastoma; molecular guided therapy; targeted agents; precision medicine neuroblastoma; molecular guided therapy; targeted agents; precision medicine
This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited (CC BY 4.0).

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Greengard, E.G. Molecularly Targeted Therapy for Neuroblastoma. Children 2018, 5, 142.

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