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Welcome to Neuren Pharmaceuticals (www.neurenpharma.com; ASX:NEU). Neuren is a Melbourne-based company at the forefront of efforts to develop effective treatments for rare, paediatric neurodevelopmental disorders.  Over the last few years, they’ve successfully steered their first drug (Trofinetide) into Phase 3 clinical trials (the last stage before approval) for the treatment of Rett syndrome.  The final …

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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.

The first biobank in Australia aiming to improve research and treatments into rare genetic diseases caused by changes to genes on chromosome 15, including Prader-Willi Syndrome and Angelman Syndrome, will be established at the Murdoch Children’s Research Institute (MCRI). 

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.