The impact of a rare disease program on the Melbourne Children’s campus

Rare Diseases Now (RDNow) was established in 2019 with funding from The Royal Children’s Hospital Foundation to provide an advanced, clinically integrated diagnostic pathway for children with rare diseases (RD). We have delivered value to children and families by providing access to frontier multi-omics technologies, working on solutions to optimise rare disease care and upskilling the workforce.  In this Grand Round we will describe the impact of our work at the individual, team and national levels and show how the Melbourne Children’s Campus is positioned as a global leader in rare disease care.​

reNEW: Transforming lives with stem cell medicine

Scientific advances now allow researchers to identify, isolate and engineer stem cells. reNEW aims to deliver treatment outcomes across the breadth of stem cell medicine – new drugs based on human stem cell models, new tissue therapies, and new cell and gene therapies. We look forward to presenting how stem cell medicine and reNEW are advancing treatments for delivery into the clinic across many currently untreatable diseases.

Medicines for ADHD: They work, but are they safe?

Meta-analyses show that medication is an effective treatment pathway in children and adolescents diagnosed with ADHD. These medications are also commonly associated with a number of side effects and risk factors for other conditions, which are less well understood by practitioners. This Grand Round intends to balance understanding of the efficacy of ADHD medication with the potential risks, and provide evidence for the safety of ADHD medication prescription in clinical practice.

Mentally healthy primary schools: A state-wide initiative to increase the capacity of schools to support children

In recent years there has been increasing policy attention paid to child mental health, at a state and national level, given the marked increase in mental health problems in children. In addition to causing distress for children and families, when mental health difficulties are not addressed in a timely way, they can become entrenched and have serious effects into adult life.

Racism and child and youth health: The public health crisis we can no longer ignore

Racism as a fundamental cause of health and health inequalities is increasingly recognised as a major public health crisis, echoing what First Nations peoples have been saying since colonisation. There is growing empirical evidence of the multiple ways in which racism impacts health and wellbeing for children and young people.

Caring for Children with Cancer Wherever They May Live: ARIA and the Global Initiative for Childhood Cancer

Is it possible, and is it ethical to resource-adapt cancer treatment developed in high income countries for the benefit of children with cancer living in low- and middle-income settings?

This is the dilemma, and the challenge, faced by health care professionals in such countries who care for 80% of the world’s children who have cancer, and where the chance of cure often remains poor. Adapting cancer treatment is a key pillar of the WHO Global Initiative for Childhood Cancer to improve the cure rates for children with cancer to 60% by 2030.