Neuren Australian Clinical Trial

Neuren is a Melbourne-based company at the forefront of efforts to develop effective treatments for rare, paediatric neurodevelopmental disorders, they plan to initiate an Australian clinical trial in Angelman later in 2021. 

Centre of Excellence

FAST Australia are committed to funding and establishing an Angelman syndrome Centre of Excellence to deliver a uniform standard of care across Australia and create infrastructure for clinical trials.

Positive Interim Phase 1/2 Data

“So far we are seeing rapid improvements in multiple areas, including some kids doing things they’ve never done before, and I don’t believe this rate of progress in development skills has been seen before in Angelman syndrome,” commented Elizabeth M. Berry-Kravis, M.D. investigator in the GTX-102 clinical study.

Angelman Syndrome Biobank

If you are a parent of a person with Angelman syndrome in Australia, the Murdoch Children’s Research Institute would like to invite you to participate in the Australian Biobank for Chromosome 15 imprinting disorders.

Sleepless in September

Fundraise for “Sleepless in September” by committing to a total of 8 hours of sleep between 6am Fri and 6:00 am on Sept 26 – 6am Sept 27th.

Roche Clinical trial

Roche and Genentech (the US member of the Roche group) have recruited the first patient in their Phase 1 clinical trial named “Tangelo” that is targeting AS with an oligonucleotide.


COVID-19 can be extra stressful for anyone with a family member with Angelman syndrome. We have compiled some information with expert advice that we will keep up to date as regularly as possible.

NEPTUNE clinical trial screening now

Two sites are open in Brisbane and Melbourne. Due to COVID-19 each site has evolving processes of scheduling screening visits, so it is important to be in direct contact with the study teams at the site closest to you.

GTX-102 Granted Fast Track Designation by U.S. FDA

“Receiving Fast Track designation for GTX-102 is an important recognition of our antisense oligonucleotide program and the promise it might offer to this patient population.” ~ Paula Evans, Chief Executive Officer, GeneTx.