Search
Research
Interleukin-1 is associated with inflammation and structural lung disease in young children with cystic fibrosisOur data associates IL-1α with early structural lung damage in CF and suggests this pathway as a novel anti-inflammatory target
Research
A novel, palatable paediatric oral formulation of midazolam: Pharmacokinetics, tolerability, efficacy and safetyWe conclude that the novel chocolate-based formulation of midazolam provides improved tolerability while remaining efficacious
Research
Predicting respiratory hospital admissions in young people with cerebral palsyMost risk factors for respiratory hospital admissions in young people with cerebral palsy are potentially modifiable
Research
Expansion of the Human Phenotype Ontology (HPO) knowledge base and resourcesThe Human Phenotype Ontology is a standardized vocabulary of phenotypic abnormalities used by researchers, clinicians, informaticians and electronic health record systems
Research
Temporal trends and socioeconomic differences in acute respiratory infection hospitalisations in children: an intercountry comparison of birth cohort studiesAdmissions for ARI were higher in Western Australia and displayed greater socioeconomic disparities than England and Scotland, where ARI rates are increasing
Research
Severity Assessment in CDKL5 Deficiency DisorderA severity assessment was rapidly developed with input from multiple stakeholders. Refinement through ongoing validation is required for future clinical trials.
Research
International experience in the development of patient-derived xenograft models of diffuse intrinsic pontine gliomaThis multi-center study provides valuable information on the success rate of establishing patient-derived pre-clinical models of diffuse intrinsic pontine glioma
Research
Planetary Health: From the Wellspring of Holistic Medicine to Personal and Public Health ImperativeHere, we explore the history of planetary health and describe its relevance to contemporary healthcare
Research
Facilitators and Barriers of Participation in “Uptime” Activities in Girls and Women With Rett Syndrome: Perspectives From Parents and ProfessionalsThis study explored facilitators and barriers to "uptime" (non-sedentary) activities in Danish girls and women with Rett syndrome
Research
A Diagnosis for All Rare Genetic Diseases: The Horizon and the Next FrontiersThere is a critical need for the field to develop and implement strategies to understand the mechanisms underlying all rare diseases and translate these to clinical care