Inaugural Australian Lysosomal Disease Summit
The Sanfilippo Children’s Foundation team were delighted to be a part of the inaugural Lysosomal Disease Summit held in Melbourne at the end of October.
The Summit was organised by Fabry Australia with the aim of bringing together clinicians, researchers and patient organisations to discuss the latest developments in research and clinical care for lysosomal storage diseases.
The agenda covered the current treatment landscape in Australia, emerging therapies, newborn screening, the value of long term data collection through registries, and the coordination of health services for people living with lysosomal diseases and the workforce to support them. We also heard about the development and application of the latest genetic therapies and new mRNA technologies for these often hard to treat conditions.
Dr Lisa Melton, Head of Research at Sanfilippo Children’s Foundation presented on the development of the Consensus Guidelines for Sanfilippo syndrome Clinical Care, which was another great opportunity to spread the word about this important resource and promote earlier diagnosis and best-practice clinical care for individuals with Sanfilippo.
The Summit provided a wonderful forum to mingle with a diverse network of researchers, clinicians, patient advocates and industry representatives from different health disciplines and locations across Australia. There were many productive sideline conversations that will undoubtedly lead to further collaboration, sharing of knowledge and resources and ultimately better outcomes for people impacted by lysosomal storage disorders.
Congratulations to the Fabry Australia team and everyone involved for this successful first event here in Australia. We look forward to many more.
