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The Kids Research Institute Australia and Perth Children’s Hospital will lead an international clinical trial of a novel drug combination they hope will increase cure rates for one of the most aggressive forms of childhood brain cancer.

A bold respiratory research project will investigate whether wearable devices could drastically change clinical care for children living with chronic diseases such as cystic fibrosis. 

Overcoming Challenges to Make Bacteriophage Therapy Standard Clinical Treatment Practice for Cystic Fibrosis Individuals with cystic fibrosis (CF)

Clinical utility of surveillance computed tomography scans in infants with cystic fibrosis Abstract Background: In cystic fibrosis (CF), irreversible

We developed a measure of hand function, and then investigated relationships between hand function, type of MECP2 mutation, age and severity of symptoms.

This study investigated the nature and prevalence of atypical pain responses in Rett syndrome and their relationships with specific MECP2 mutations.

We investigated whether children who are heavier at birth have an increased risk of type 1 diabetes

To develop guidelines for the clinical management of scoliosis in Rett syndrome through evidence review and consensus expert panel opinion.

In China, there are severe unmet medical needs of people living with rare diseases. Relatedly, there is a dearth of data to inform rare diseases policy. This is historically partially due to the lack of informatics infrastructure, including standards and terminology, data sharing mechanisms and network; and concerns over patient privacy protection.

Severe dengue is a life-threatening complication; rapid identification of these cases, followed by adequate management is crucial to improve the clinical prognosis. Therefore, this study aimed to identify risk factors and predictors of severe dengue.