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Showing results for "clinical trials"
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Chemosensitisation of medulloblastoma and pineoblastomaRaelene Nick Endersby Gottardo BSc (Hons) PhD MBChB FRACP PhD Brainchild Fellow; Co-Head, Brain Tumour Research Head of Paediatric and Adolescent
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Vitamin D deficiency and the lung: Disease initiator or disease modifier?Vitamin D deficiency is a global public health problem and has been associated with an increased incidence and severity of many diseases including diseases...
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Radiographic and visual response to the type II RAF inhibitor tovorafenib in children with relapsed/refractory optic pathway glioma in the FIREFLY-1 trialDue to their anatomical locations, optic pathway gliomas (OPGs) can rarely be cured by resection. Given the importance of preserving visual function, we analyzed radiological and visual acuity (VA) outcomes for the type II RAF inhibitor tovorafenib in the OPG subgroup of the phase 2 FIREFLY-1 trial.
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"Pre-emptive strike"-the case for early treatment of hepatic sinusoidal obstruction syndrome with defibrotideWe describe the case of a child with a right 11th rib primitive neuroectodermal tumor who developed HSOS following focal radiotherapy and actinomycin-D treatment
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Metabolic and Psychological Impact of a Pragmatic Exercise Intervention Program in Adolescent and Young Adult Survivors of Pediatric Cancer-Related Cerebral InsultAdolescent and young adult survivors of childhood brain tumors and cranial irradiation should be screened for metabolic and psychological well-being
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Gene Expression Analyses of the Spatio-Temporal Relationships of Human Medulloblastoma Subgroups during Early Human NeurogenesisMedulloblastoma is the most common form of malignant paediatric brain tumour and is the leading cause of childhood cancer related mortality.
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ONC201 in Combination with Paxalisib for the Treatment of H3K27-Altered Diffuse Midline GliomaDiffuse midline gliomas (DMG), including diffuse intrinsic pontine gliomas (DIPG), are the most lethal of childhood cancers. Palliative radiotherapy is the only established treatment, with median patient survival of 9 to 11 months. ONC201 is a DRD2 antagonist and ClpP agonist that has shown preclinical and emerging clinical efficacy in DMG.
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Trial refresh: A case for an adaptive platform trial for pulmonary exacerbations of cystic fibrosisThis review will discuss the necessary steps required for a Bayesian adaptive platform trial to optimize treatment of pulmonary exacerbations of cystic fibrosis
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Assessing the effect of meningitis prevention and treatment.In high-income countries serious bacterial infections such as meningitis are uncommon, but their severity has led to prompt adoption of vaccines for...
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Polyamine depletion limits progression of acute leukaemiaCancer cells are addicted to polyamines, polycations essential for cellular function. While dual targeting of cellular polyamine biosynthesis and polyamine uptake is under clinical investigation in solid cancers, preclinical and clinical studies into its potential in haematological malignancies are lacking. Here we investigated the preclinical efficacy of polyamine depletion in acute leukaemia.