In the last decade, we have seen genetic therapies leap from the benchtop into the neuromuscular clinic. This process is most dramatically illustrated by spinal muscular atrophy (SMA), which was until recently the most common genetic cause of death in infancy.
Healthy Trajectories is proud to bring a celebration of the International Day of People with Disability (IDPwD) to Grand Rounds at The Royal Children’s Hospital. In line with our vision for an inclusive Australia, we will share what we and others around the Campus have been doing to authentically involve young people growing up with a disability, and their families, as partners in research.
Olivia is a 14-year-old girl from rural NSW who was diagnosed with Pre B Acute Lymphoblastic Leukemia (ALL). The treatment is long and arduous, but if treated immediately has a 90 percent survival rate. Without treatment she will die within four weeks. Olivia has other conditions including epileptic encephalopathy (DEE), a severe intellectual disability, global developmental delay, communication difficulties, drug-resistant seizures and behavioural difficulties. The cancer therapy would require Olivia to have over 50 general anaesthetics as she won’t accept treatment without being restrained.
What does disability care for children and young people look like in 2023, and what could it be like in the future? In 2023 it is more holistic, more funded, more early intervention, more complex, and more positive for children and their families.
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.
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.
Our Healthy Trajectories research is done in partnership with consumers, clinicians, and researchers with diverse disciplinary expertise. Our goal is to contribute evidence to rapidly improve the health, wellbeing, and participation of those with child-onset disability and their families. We will only be able to ensure that people with disability can participate as equal members of society if we collaborate effectively across disciplines and sectors: we invite your involvement.
The Melbourne Children’s Campus is a global leader for longitudinal and life course data. Being one of the few centres worldwide with such extensive longitudinal cohort studies, we are positioned to address an array of questions central to early life health and development and its progression throughout the life span.
Sophie Deane is a 20-year-old young woman with some pretty big ideas about what she would like to do with her life. She has just finished school and is keen to get on and find a job, move out of home, travel – and maybe even get married. She would like the chance to tell you more about those hopes and dreams.
A Stepped Care Approach to Developmental Care is a Royal Children’s Hospital Foundation funded project which aims to ensure that children referred with a developmental concern are seen by the right person in the right place at the right time. To achieve this a new Allied Health centralised intake team apply a stepped approach that identifies the child’s needs and pathway.
