The conference will be an opportunity for the Angelman Community to come together and connect face-to-face, while learning about the latest research, clinical trials, therapies, practical and family support, NDIS, and much more. The conference will be accessible, and everyone is welcome to attend.
Conference – Friday and Saturday 8:30-5pm. $180
The conference registration fee includes attendance & activities for individuals for Angelman syndrome on both days, the full registration cost may be claimed through NDIS Capacity Building funds by referencing Day 2 of the program. Download the flyer here for more information.
Other activities/events
Conference room rates are available for the Hotel Grand Chancellor, contact us at info@cureangelman.org.au for the code.
Draft Program, subject to change
Opening, welcome to country | |
Keynote – science overview. Angelman syndrome pipeline | Dr Barbara Bailus, Keck Graduate Institute California |
Research | |
Therapeutics for all genotypes | Dr Randal Moldrich, FAST SAB chair |
UPenn gene therapy program | Dr James Wilson, UPenn (teleconf) |
Hemapoetic stem cells & Enzyme Replacement Therapy | Dr Barbara Bailus |
PTC – Program update | PTC Therapeutics |
CRISPR for Angelman syndrome | Dr David Segal, UC Davis California |
Questions? | Panel |
Break | |
Australian research | |
Australian considerations to accessing trials and potential treatments | Anna Walsh FAST Australia Board |
Microbiome in Angelman. What we do & dont’ know | Drs Jessica Duis Colorado Children’s Hospital/Honey Heussler Children’s Health Queensland |
Wearable technology as a biomarker | Dr Claudine Kraan, Murdoch Children’s Research Institute |
Newborn screening | Dr David Godler, Murdoch Children’s Research Institute |
Search & Rescue – Australian team and project | Panel TBA |
Clinic network update & panel | |
Angelman syndrome Standard of care | Drs Jessica Duis, Anjali Sadhwani (Boston Childrens Hospital), Honey Heussler |
Benefits of long term clinics (Boston) Experiences. Wins & Losses | Dr Wen Hann Tan, Boston Children’s Hospital |
intro to the clinics & clinicians and nurses | |
Lunch | |
Clinical trials – current | |
Experiences & insights into running clinical trials for AS in the US | Dr Elizabeth Berry Kravis, Rush University Hospital |
Upstream and downstream therapies & the importance of trials | Chloe Simons, FAST Chief Science Officer |
Clinical Trial update | Neuren Pharmaceuticals |
New Program Alogabat | Roche – Speaker TBA |
Break | |
Antisense oligonucleotides (ASOs) | |
Ultragenyx program update – GTX-102 | TBC |
Ionis program update – Halos | Dr Becky Crean, Ionis Pharma |
Roche – Tangelo | Roche – Speaker TBA |
Questions | Panel |
Close |
Advocacy & Support – Day 2 | |
Coffee from 8:30 – Commences at 9.00am | |
Opening | Fiona Lawton, ASAA President |
Introduction to the ASAA Committee | Fiona Lawton, ASAA President |
Systemic Advocacy | |
Rare Voices Australia – Rare Disease Advocacy update | Louise Healy, RVA |
National Patient Organisation Network Australia | Kate Holliday, NPON |
Disability Royal Commission – What we heard | Peter Young, DRC |
Healthcare for People with Intellectual Disability | TBC |
Communication Friendly Hospital Initiative | Bethany Hooke, Queensland Children’s Hospital |
Palliative Care for People with Intellectual Disability | Dr Janelle Weiss 3DN |
Questions? | Panel |
Break | |
Support | |
Navigating NDIS (Panel) | Fiona Lawton/Leticia Grant/Qld Dept Rep |
Communication through the ages – It’s never too late to start | Sheree Brisbane, SpeechEase Speech Therapy |
Disability Done Differently – Building an Inclusive Communtiy | Jake Cowie, National Support Manager What Abilty |
The Many Faces of Grief | Letitia Grant, ASAA |
My sister, My brother, My Lilfe ! | Panel Session – Dr Anjali Sadhwani, Boston Children’s Hospital with Angelman siblings |
Angelman syndrome – Beyond the obvious – the weird and not-so-wonderful | TBC |
Estate Planning for The Present and The Future | Anthony Vidray, AV Chartered Accountants |
Questions? | Panel |
Lunch | |
Breakout Sessions – Age Group Intensive Q&A (Various leads) | |
Break out 1 – Early Years | |
Sleep Management in the Early Years | Dr Honey Heussler, Queensland Children’s Hospital |
Intensive Model of Therapy for Neurological Disorders | TBC |
School choices – Where to go and how to know? | Diana Backhouse |
Break out 2 – Teen Years | |
Puberty, Hormones, Anxiety and Angelman syndrome | Dr Jessica Duis, Colorado Children’s Hospital |
Positive and Active Behaviour Support | Christine Creech, Positive Behaviour Support Practitioner, Positive Moods |
Turning 16 – A Major Milestone Transition – Centrelink, School & Health | Fiona Lawton, Neu Pathways Australia |
Break out 3 – Adult Years | |
Guardianship – The Pro’s and Con’s | Letitia Grant |
Independent Living – Creating a Good Life/Microboards | Jacqui Mills, Microboards Australia; Is There A Better Way |
Customised Employment Options — What could this look like? | TBC |
Afternoon Tea | |
Final session | Breakout Session Summaries |
ASAA Strategic Direction | |
International Angelman Day Recognition | |
Close |
11am-4:30pm. Sponsored by FAST Australia. For professionals only please.
Free to attend, ticket link to register for catering will be live soon.
What the AS space looks like right now | Dr Barbara Bailus, Keck Graduate Institute California |
Lightening rounds for clinical trials | Pharma updates (Neuren, Roche, Ionis, Ultragenyx, PTC) |
Unexpected challenges of running trials | Dr Elizabeth Berry Kravis, Rush University Hospital Chicago |
Challenges with outcome measures/ABOM | Dr Barbara Bailus, Keck Graduate Institute California |
Meaningful change thresholds in AS | Dr Anjali Sadhwani, Boston Childrens Hospital |
Wearable technology as a biomarker | Dr Claudine Kraan, Murdoch Childrens Research Institute |
Panel/Questions | |
Lunch | |
Introduction to session | |
Parent reported challenges | TBD |
Insights from long term clinics | |
Anxiety and behaviours in clinic | Dr Anjeli Sadwani, Boston Childrens |
Coordinating disciplines & responding to kids in crisis | Dr Jessica Duis, Childrens Hospital Colorado |
Data collection & Natural History | Dr Wen Hann Tan, Boston Childrens Hospital |
Break | |
How to address underdiagnoses (Search & Rescue) – think tank | |
Roundtable – Perfecting an Australian model for clinics/Centres of excellence and clinical trial centres |