Biography

Associate Professor Kim Hemsley has devoted the past 19 years to establishing a world-leading research group charged with elucidating the pathogenic basis of disease and developing therapies for Sanfilippo Syndrome, a currently untreatable childhood-onset form of dementia. Whilst this condition is rare, it is one of ~70 genetic diseases that cause childhood dementia. 1 in 2800 children/young people experience childhood dementia (~700,000 worldwide). Only 5% have treatments. 

In collaboration with other leading researchers, A/Prof Hemsley has directed NHMRC- and Industry-funded pre-clinical evaluation studies with translational outcomes: Phase I/II or II/III studies of three novel treatments in Sanfilippo patients.

A/Prof Hemsley's innovative research program aims to a) develop tools for predicting the rate of symptom-onset/disease-progression in Sanfilippo syndrome; b) determine the basis for symptom-generation, thereby providing a rational target for therapies; c) devise and evaluate novel treatments and d) provide biomarkers for monitoring therapeutic efficacy.

This will enable the implementation of existing technologies for newborn screening for childhood dementia in Australia, therapeutic intervention at the earliest possible age/disease stage, and the greatest improvement in patient quality and quantity of life.

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