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.
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.
“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.
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.
The first patients have been enrolled in the Genetx (Ultragenyx Pharmaceutical Inc) KIK-AS trial in the United States.
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. https://sleeplessinseptember.gofundraise.com.au/
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.
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.
“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.