Rapid evaluation and subsequent regulatory approval of new drugs are critical to improving
survival and reducing long-term side-effects for children and adolescents with cancer. The …

Abstract The first (2017) and sixth (2021) multistakeholder Paediatric Strategy Forums
focused on anaplastic lymphoma kinase (ALK) inhibition in paediatric malignancies. ALK is …

The seventh multi-stakeholder Paediatric Strategy Forum focused on chimeric antigen
receptor (CAR) T-cells for children and adolescents with cancer. The development of CAR T …

Simple Summary Precision medicine is a revolutionary new way to deliver cancer treatment
by targeting specific genetic changes of the cancer of the individual child with the goal of …

As the mitogen-activated protein kinase (MAPK) signalling pathway is activated in many
paediatric cancers, it is an important therapeutic target. Currently, a range of targeted MAPK …

Abstract The eighth Paediatric Strategy Forum focused on multi-targeted kinase inhibitors
(mTKIs) in osteosarcoma and Ewing sarcoma. The development of curative, innovative …

DNA damage response inhibitors have a potentially important therapeutic role in paediatric
cancers; however, their optimal use, including patient selection and combination strategy …

Based on biology and pre-clinical data, bromodomain and extra-terminal (BET) inhibitors
have at least three potential roles in paediatric malignancies: NUT (nuclear protein in testis) …

Cancer is the second biggest cause of death in children in the US. With the development of
chemotherapy, there has been a substantial increase in the overall survival rate in the last …

The investment in the pharmaceutical development of medicines for paediatric use
represents a minority when compared to that one made for adult population. Which reasons …