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.
“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.
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.