All News Posts 

Today Neurogene announced a new development program for Rett syndrome, NGN-401. NGN-401 is an MECP2 gene therapy candidate using Neurogene’s novel EXACT technology, which was developed in collaboration with the University of Edinburgh. This work at the University of Edinburgh is led by Stuart Cobb, Ph.D., who has been researching…

  Dear RSAA  Members Our 32nd RSAA  AGM  will be held on  Saturday 11th December 2021 at 4.00 pm (AEDT), 3.30 pm (SA Time), 3.00 pm (QLD Time) & 1.00 pm (WA Time).   As COVID 19 prevents us from meeting in person, the  AGM  will be held online and…

              Background. As noted in previous articles now on the RSAA website, gene therapy for Rett Syndrome represents a potential cure. Other potential strategies are being explored but gene therapy probably represents the strategy most likely to reach the clinic first. Many of these…

Support the Rett Syndrome Association of Australia during the Rett Syndrome Awareness month of October Follow this link to buy tickets. RaffleTix The prize is a $500 Bunnings Voucher To be drawn on Sunday 31st October     

WATCH: Gene Replacement for Rett is Closest to the Clinic Gene therapy is one of six strategies we're pursuing at RSRT that attacks the underlying genetic error in Rett syndrome. Gene replacement is the closest to clinical trials and, if those trials are successful, the clinic. Learn more from one…

  Novel clinical evaluation strategies are needed to fulfil the potential of targeted therapies for rare neurodevelopmental disorders.   An article by Michelle L. Krishnan1,14, Elizabeth Berry-Kravis2, Jamie K. Capal3, Randall Carpenter4, Paul Gringras5, Joerg F. Hipp1, Meghan T. Miller1,15, Ana Mingorance6, Benjamin D. Philpot7, Mathew T. Pletcher1,16, Alexander Rotenberg8,…

This brief article is a follow-up to previous articles now on the RSAA website (https://rettaustralia.org.au/blog/novartis-and-gene-therapy-to-treat-rett-syndrome/ AND https://rettaustralia.org.au/blog/adeno-associated-virus-aav-as-a-delivery-vehicle/). Click here to read the latest article

A recent article noted that recombinant AAV production facilities worldwide were currently committed to covid-19 vaccine production and that this might delay any proposed clinical trials to treat patients with Rett Syndrome. This short article provides some background on AAV and the difficulties in scaling up production to permit an…

A talk by Tracey Campbell, Abigail Davison-Hoult and Gill Townend 18 June 2021, 5:00 am AEST REGISTRATION IS FREE - Book Your Place Here About this talk Join us for the fifth Discussion Group of 2021. In this session we will be focussing on Kate Ahern and Judy Lariviere's talks on the impact…

The Rett Syndrome Research Trust, USA (RSRT) has continued to broadcast webinars which are designed to introduce researchers with an interest in developing a cure for Rett Syndrome, and present details of progress.  As noted in previous articles, RSRT provides financial support to researchers in six major areas of research,…