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.
In this Grand Round, Associate Professor Mark Mackay will present advances in acute stroke care, through implementation of standardised paediatric “Code Stroke” protocols, to facilitate rapid diagnosis and treatment of stroke, and how the Melbourne Children’s campus is leading the way in changing practice nationally and improving outcomes for the youngest stroke survivors.
How are artificial intelligence, big data, and minimally invasive technologies changing the way we diagnose and treat children with drug-resistant epilepsies?
The approach and attitude towards Functional Neurological Disorders (FND) has changed over the last decade and anecdotally at least, we seem to be seeing more cases in our day-to-day practice. Patients with functional symptoms present to many disciplines and functional symptoms can co-exist with organic pathology. There is now evidence to show earlier diagnosis and treatment can lead to more positive outcomes.
Among individuals with cerebral palsy (CP), rehabilitation needs and treatment have been established, but are mostly focused on the pediatric population. Evidence demonstrates that the number of adults who live with CP and other childhood-onset disabilities is increasing, and that they experience significant physical and mental health inequalities as they grow up.
Demyelinating disorders of childhood include a broad range of conditions such as acute disseminated encephalomyelitis, optic neuritis, transverse myelitis, and multiple sclerosis (MS). When children present with a first demyelinating event, parents often ask the question: ‘Does my child have MS’?
The Melbourne Children’s Campus is seeing an increase in the number of clinical trials of gene therapies, especially in vivo viral vector therapies. With some of these therapies now approved by the TGA, there is an expectation by families that these therapies will be available at the Children’s.
The Complex Movement Disorders program at the Royal Children’s Hospital commenced in 1997 and has evolved since then. It is currently funded by RCH Foundation and the fundraising efforts of A/Prof Andrew Kornberg’s Fly for the Kids event in 2017. The program allows coordinated multidisciplinary assessment and management of children with movement disorders and associated neurodevelopmental comorbidities.
There has in recent years been a rapid increase in the number and complexity of clinical trials and novel therapies for neurogenetic conditions. Many of these conditions are individually rare, but their impact upon affected children and their families may be very severe. While the increasing awareness and availability of new treatments brings great hope and excitement for all involved in care of these children, it also presents significant challenges for clinicians and for patients and their families.
Electroencephalography (EEG) has a long history at The Royal Children’s Hospital. From its beginnings in the pre-imaging era as simple paper recordings of brain waves in children with various neurological disorders, EEG has evolved to a specialised discipline within neurophysiology, drawing on the many advances in clinical neurology, computerisation and neuroimaging.
