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Research

International experience in the development of patient-derived xenograft models of diffuse intrinsic pontine glioma

This multi-center study provides valuable information on the success rate of establishing patient-derived pre-clinical models of diffuse intrinsic pontine glioma

Research

Maternal high fat diet compromises survival and modulates lung development of offspring, and impairs lung function of dams

While maternal high fat diet compromised litter survival, it also promoted somatic and lung growth (increased lung volume) in the offspring

Research

Interleukin-1 is associated with inflammation and structural lung disease in young children with cystic fibrosis

Our data associates IL-1α with early structural lung damage in CF and suggests this pathway as a novel anti-inflammatory target

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A novel, palatable paediatric oral formulation of midazolam: Pharmacokinetics, tolerability, efficacy and safety

We conclude that the novel chocolate-based formulation of midazolam provides improved tolerability while remaining efficacious

Research

Research priority setting in childhood chronic disease: a systematic review

Research priority setting activities in paediatric chronic disease cover many discipline areas and have elicited a broad range of topics

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Expansion of the Human Phenotype Ontology (HPO) knowledge base and resources

The Human Phenotype Ontology is a standardized vocabulary of phenotypic abnormalities used by researchers, clinicians, informaticians and electronic health record systems

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Predicting respiratory hospital admissions in young people with cerebral palsy

Most risk factors for respiratory hospital admissions in young people with cerebral palsy are potentially modifiable

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A group randomized controlled trial evaluating parent involvement in whole-school actions to reduce bullying

Whole-school capacity-building intervention in early and middle childhood can improve the likelihood and frequency of positive parent–child communication about bullying

Research

Building the repertoire of measures of walking in Rett syndrome

This study aimed to determine measurement properties of a modified 2MWT and a modified Rett syndrome-specific FMS-RS in Rett syndrome.

Research

Orthopaedic issues in Rett Syndrome

This chapter reviews the prevalence, characteristics, and clinical management of orthopedic problems in RTT.