WATCH: Advanced Therapeutics Webinar Series 10: a look at how gene therapy is changing the game for children with SMA

11 November 2022
Webinar series
Photo of a sleeping baby in relation to Kids Research Spinal Muscular Atrophy trials and success

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 gene therapy neuromuscular service, as well as milestones from patients.

Up until recently, SMA has been the leading genetic cause of infant death in Australia. However, in recent years our researchers and clinicians have made enormous progress in the early identification and treatment of the condition, and with the combination of newborn bloodspot screening for SMA and the success of novel genetic therapies, have been able to change the game for these patients.

In the 10th instalment of our series, experts will take the audience through the journey of a patient living with SMA, including the newborn bloodspot screening, diagnosis, treatment delivery, clinical outcomes and applications for the future.

Speaker profiles:

A/Prof Michelle Farrar


A/Prof Farrar is an Associate Professor in Paediatric Neurology at UNSW Sydney and specialist child neurologist at Sydney Children’s Hospital, leading the Neuromuscular clinical and research programs. She is a leading researcher in paediatric neuromuscular and rare neurogenetic disorders, successfully translating research to improve health practice and outcomes for patients, their families and the community.

A/Prof Farrar’s team is focused on developing and translating innovative ways of providing the best possible care from the time of diagnosis, through treatment and beyond to people with neuromuscular, neurodevelopmental or rare neurogenetic diseases. The research program also integrates interdisciplinary genomics research, together with community and consumer involvement.

A/Prof Farrar also has a strong commitment to improving the practice of medicine, thus connecting her research achievements to her activities in medical education and community and consumer engagement.

Dr Sandi Kariyawasam


As a clinical researcher Dr Kariyawasam combines her role as a Staff Specialist with her research, striving to improve outcomes for children with neurogenetic disease. As a primary investigator of the first newborn screening (NBS) program for spinal muscular atrophy (SMA) in Australia, she has established the utility, feasibility and pathways to sustainability for this model of screening.

Her work to assess implementation, diagnostic and clinical pathways for NBS for SMA, the public acceptability of the program and short-term health and psychosocial outcomes have formed the basis for a submission statement to the National NBS Standing Committee and served to promote the national adoption of NBS for SMA as of 2021.

Ms Deshina Naidoo


Deshina is a Project Pharmacist for the Sydney Children’s Hospitals Network working within the Clinical Governance Unit (CGU) and Children’s Hospital Westmead (CHW) Pharmacy Department. She has worked as a Clinical Pharmacist at both adult and paediatric public hospital facilities with experience in neurology, surgery, orthopaedics and critical care.

She is the Executive Officer of the National Paediatric Medicines Forum under CGU as well as lead pharmacist in the successful implementation of innovative smart pump drug libraries at CHW. She currently supports the pharmaceutical management of advanced therapeutics including preparation and delivery of gene therapy products and clinical trials at CHW.

Deshina has an interest in novel therapies in paediatrics and looks forward to developing paediatric resources and improving awareness in the evolving advanced therapeutics space.

Ms Sandra Charlton


Sandra has a strong background in Paediatric nursing with postgraduate qualifications in Paediatric and Neonatal Intensive Care. She holds a Master of Philosophy with a strong research focus. Her expertise ensures provision of quality care, risk minimisation and achieving desired patient outcomes for children who receive advance therapeutics including gene therapy. 

Sandra has been pivotal in establishing and expanding the Gene therapy program at SCHN. She is a key member on advisory committees and working groups within the SCHN and for external stakeholders, with a key focus on setting up a National Gene Therapy service for children who have Spinal Muscular Atrophy.

Sandra’s vast experience has allowed her to provide clinical leadership, education, support, consultancy in the development, implementation and evaluation of nursing care delivery that is patient focused, improves clinical care and patient outcomes.




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