A GAME CHANGER FOR GENE THERAPY FOR RETT SYNDROME.

              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…

RETT SYNDROME AWARENESS MONTH ANNUAL RAFFLE #2

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     

Gene Replacement for Rett Syndrome 101

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…

Clinical Trial Strategies for Rare Neurodevelopmental Disorders

  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,…

Progress of gene therapy to treat Rett Syndrome

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

Adeno-associated virus (AAV) as a delivery vehicle. Production delays.

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…

Discussion Group – What Else Affects Communication

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…

Staying Resilient (self) Discussion Group

Start date & time  29 June, 2021 - 10:00 End date & time  29 June, 2021 - 11:00 Venue  Online Status  View event details for more information. Rett UK are pleased to offer to all families a series of webinars from Gooding & Wood to help Rett families navigate through…
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