News & Events

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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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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Gene therapy
At four months of age Sophia is reaching all her developmental milestones. She demonstrates good head control while sitting supported and can even roll from her tummy to her back. Her parents Kaitlin and Max have not witnessed any decline in Sophia’s physical development and for this they are extremely grateful to the Paediatric Neurology team at Sydney Children’s Hospital, Randwick (SCH)....
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International Women's Day
Wendy Gold is an Associate Professor at the University of Sydney in the School of Medical Sciences, Faculty of Medicine and Health and a Neurobiologist based in the Kids Neuroscience Centre, Kids Research at The Children’s Hospital at Westmead. She teaches third year Medical Science students on the subject of translating medical discoveries into the clinic and leads a research team who...
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CMT Guidelines
The first ever clinical practice guidelines for children with Charcot-Marie-Tooth disease (CMT) have been published after a decade of work by an international team. CMT is a group of degenerative genetic disorders affecting the peripheral nervous system, the nerves stretching from the spinal cord to the muscles. It’s a lifelong condition which usually starts in childhood and progresses into ad...
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Rare Disease Day
The benefits of a new precision medicine initiative, being piloted at Sydney Children’s Hospitals Network (SCHN), are already being seen in children and families with rare genetic diseases. The program, Gene2Care, aims to improve the standard of care, including enhanced diagnoses and access to management and treatment. The family-centred program, funded by Luminesce Alliance, combines the effo...
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Genetic Testing
A new test for genetic diseases is successfully providing the sought-after answers for families of sick children. RNA diagnostic testing has already achieved a diagnosis for more than 70 families, with hopes it can be implemented in everyday clinical practice. Current methods using DNA sequencing are only able to diagnose half of the children and families with suspected genetic disorders. Prof...
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Gene Therapy
Children with genetic diseases and cancers will be given faster access to life-saving gene and cell therapies through a new commercial scale viral vector manufacturing facility in Western Sydney. The NSW government has called on industry and investors to submit expressions of interest to support the growth and operations of the facility, to be built in the Westmead Health and Innovation Distri...
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