Strengthening study start-up for novel therapies

The Advanced Therapeutics Medical Lead is a new position that has been established to support teams to deliver effective, innovative and often complex treatments and clinical trials to more children who need them.

Paediatric Neurologist and Clinical Trials Lead, Dr Michelle Lorentzos was appointed to the new role working with the Kids Advanced Therapeutics team.

“In this new position, I am looking forward to better addressing some of the delays in getting new trials and treatments up and running,” said Dr Lorentzos.

“There may be ways we can streamline some processes, and do this in a way that is guided by what children and families want, and I am excited about that.”

Building improved purpose-built clinical research facilities

The new purpose-built Clinical Research Centre is larger and facilitates a seamless workflow for teams to deliver a comprehensive range of treatments and interventions within the same space.

“The new space is comfortable, with a clean layout, making our visits smoother and less stressful,” said Byrony, mum to John, who participates in a clinical trial requiring him to attend monthly appointments.

Australian-first gait analysis lab changing how we provide care

The upgraded Paedaitric Gait Analysis Lab at The Children’s Hospital at Westmead features new state-of-the-art technology to help clinicians and researchers assess the physical capacity of children and young people with movement disorders.

The clinical space is larger and houses an Australian-first 30 metre purpose-built, distraction free walking track.

Watch its feature on 9News and how the new space is impacting young people like Ali.

Bridging the knowledge gap for advanced therapies

The Kids Advanced Therapeutics program have developed resources and programs that support patients, staff and communities in educating about advanced therapies.

A gene therapy factsheet and gene therapy educational videos provide a family-friendly way to break down complex information around gene therapy treatment and management in an easily accessible way for patients, parents and carers.

In collaboration with hospital Neurology Departments and the Brain Aid project, the program further contributed to the Advanced therapies digital handbook, a comprehensive resource helping parents and carers understand some of the advanced therapies used to treat some childhood rare conditions.

The team have additionally facilitated support for clinical staff and researchers through the launch of the AAV Gene Therapy Education program and its regular research seminars and webinars.

All of these initiatives were kindly supported by the Sydney Children's Hospital Foundation, Luminesce Alliance, Cerebral Palsy Alliance and Roche Australia.