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…

Novartis and gene therapy to treat Rett Syndrome.

Novartis, a multinational biotechnology company, issued a statement in August 2020 to confirm full commitment to their gene therapy program for Rett Syndrome. This was followed by a press release from the company in March 2021 which reported recent results of gene therapy to treat patients with spinal muscular atrophy…

New hope for girls with Rett Syndrome

ANAVEX®2-73 - A drug used to help adult Alzheimer’s patients may offer hope to young girls with Rett syndrome. In a new clinical trial being led by The Children’s Hospital at Westmead, doctors are testing whether ANAVEX®2-73 can help improve some of the devastating features of Rett syndrome including their…
Share this story