News & Events

Photo of a sleeping baby in relation to Kids Research Spinal Muscular Atrophy trials and success
Webinar series
Advanced Therapeutics: No longer science fiction – a look at how gene therapy is changing the game for children with SMA In our upcoming webinar, we will be visiting the topics of newborn bloodspot screen and genetic therapies in the context of treating Spinal Muscular Atrophy (SMA). We will hear from a panel of speakers who will discuss our centres success in establishing a multidisciplinary...
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Cancer research
A researcher’s vision to analyse genes of young cancer patients to personalise their treatment has now become a virtual reality. Twenty years ago, Associate Professor Daniel Catchpoole developed an idea to examine complex data and deliver precision medicine through an immersive experience. Now, a prototype for ‘Virtual Reality for the Observation of Oncology Models’, VROOM for short, has be...
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Respiratory research
Lockdowns led to a dramatic decrease in the number of young people taken to Sydney hospitals for their asthma during the pandemic. These are the findings from a new study, published in Springer Nature, investigating the impact of the three COVID-19 waves in NSW on paediatric asthma-related hospital presentations. The research found a significant decline in presentations while stay-at-home rest...
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Webinar series
In the 8th episode of our Advanced Therapeutics Webinar series we revisited the topic of ocular gene therapy with our biggest panel of speakers this year. Since LUXTURNA’s success as a world-first gene replacement therapy for an RPE65-related retinal vision loss early last year, our researchers have made great strides in paving the way for the treatment of patients with all kinds of...
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Viral Vector Facility
Kids Research welcomes the NSW government’s funding boost for biomedical research, putting the state at the forefront of cutting-edge health treatments. The Perrottet government has secured $270 million, announced in the state budget, to go towards two new facilities; the Viral Vector manufacturing facility at Westmead and the Sydney Biomedical Accelerator Complex in Camperdown. Premier Domini...
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Gene therapy
World-leading gene therapy experts at Sydney Children’s Hospitals Network (SCHN) have shared their knowledge and experience on a global stage. Researchers, Professor Ian Alexander and Associate Professor Michelle Farrar have spoken at separate conventions in California, after being honoured with an invitation to the events. The pair led the only Australian clinical trial of gene th...
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COVID-19 study
A new study has found the number of Australian adults infected with COVID-19 at the end of February, was at least twice as high as cases reported to authorities. At least 17 per cent of adults are estimated to have recently been infected with SARS-CoV-2, the virus that causes COVID-19, with the majority of these infections believed to have occurred during the Omicron wave. This result, rele...
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World-first SMA treatment
Babies diagnosed with Spinal Muscular Atrophy (SMA) at birth who received life-changing gene therapy are now celebrating their first birthday symptom-free or with minimal symptoms thanks to Sydney Children’s Hospitals Network’s (SCHN) world-first clinical trial. The SPR1NT trial, published in the prestigious journal Nature Medicine (two copies of SMN2 and three copies of SMN2), successfull...
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Webinar series
In the 7th instalment of our Advanced Therapeutics Webinar series we focused on ethical considerations in rare disease clinical trials. As new treatments become available, and in populations without prior access, concerns around equity and suitability arise. Physicians, patients and their families can be faced with a situation where there are more patients than clinical trial spots. So, who...
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Pandemic trends
The number of teenage girls who presented to NSW Emergency Departments (ED) for self-harm or suicidal ideation has dramatically escalated during the pandemic, following a steady increase over the past decade. A new study has investigated whether the COVID-19 pandemic has worsened trends in self-harm presentations in children and young people, which have increased globally over the last 10 year...
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