Lysosomal Disease Patient Conference
The Lysosomal Disease Patient Conference will be held on Saturday, 19 October, 2024.
The 2024 Patient Conference will be presented by Fabry Australia, in conjunction with the Australian Pompe Association, the Sanfilippo Children’s Foundation and the Gaucher Association of Australia & New Zealand.
Preliminary Topics and Speakers
Saturday, 19 October, 2024
| 8:00 | Registration | |
| 8:50 | Welcome | |
| 9:00 | Jo Watson Deputy Chair, Pharmaceutical Benefits Advisory Committee (PBAC) Canberra, ACT, Australia | The role of the LSDP and PBAC, with rare disease medicines. |
| 9:30 | Donna North Research Co-ordinator Royal Melbourne Hospital Melbourne, VIC, Australia | Understanding Clinical Trials. |
| 10:00 | Ying Sun Cincinnati Children’s Hospital Cincinnati, OH, United States | Emerging Treatments for Unaddressed Needs in Lysosomal Storage Disorders. |
| 10:30 | Exhibits Open | Morning Tea |
| 11:00 | Heidi Peters Royal Children’s Hospital Melbourne, VIC, Australia | Pain and Lysosomal Diseases. |
| 11:30 | Cate Clark | The Mental health burden of rare disease. |
| 12:00 | Lunch with Exhibitors | |
| 13:00 | Uma Ramaswami Royal Free Hospital London, United Kingdom | What are biomarkers and how are they used? |
| 13:30 | Adam P. Vogel University of Melbourne Melbourne, VIC, Australia | Speech and language function in Lysosomal diseases. |
| 14:00 | Dianne Wallyn, Fabry Australia Jeff Buckell, Pompe Australia Gidon Goodman, Gaucher Association of Aust and NZ Sarah Beattie-Stevens, Sanfilippo Children’s Foundation | What I would tell my younger self? A patient’s perspective. |
| 15:00 | Exhibits Open | Afternoon tea |
| 15:30 | Tess Aitken Royal Melbourne Hospital Melbourne, VIC, Australia | The impact of Fabry disease on bone density and treatment effects. |
| 16:00 | Gail Hilton Head of Strategy at Childhood Dementia Initiative Sydney, New South Wales, Australia | Navigating health and social care systems – family experiences. |
| 16:30 | Ineka Whiteman Head of Research and Medical Affairs BDSRA Foundation Principal Scientific Consultant Beyond Batten Disease Foundation Mia Holloway Team Leader, HammondCare Dementia Support Australia | Practical management approaches to behaviours and psychological symptoms in childhood dementias caused by lysosomal disease and related conditions. |
| 17:00 | Wrap up | |
| 17:15 | Collaboration Reception | Posters and Exhibits Open |
| 18:15 – 23:30 | Celebration Dinner | All Summit and Patient Conference participants are invited to relax and reconnect at the Collaboration Reception and Fabry Australia’s 30th Anniversary Celebration Dinner |
The 2024 Lysosomal Disease Patient Converence will be held at the Pullman & Mercure Melbourne Albert Park in Melbourne, Australia. Click here to make a hotel reservation.
Patient Conference Sponsors:
The Patient Conference is supported in part by funding from Industry Groups. who have had no input into the concept, planning, design, content or delivery of this activity.
More information will be available soon on the Fabry Australia website at https://www.fabry.com.au.