Anavex Life Sciences Corp. announced the completion of the ANAVEX®2-73 (blarcamesine) U.S. Phase 2 study in Rett syndrome. The Company expects to announce topline results from this study in the next quarter. ANAVEX®2-73 is an orally available, small-molecule activator of the sigma-1 receptor which, data suggest, is pivotal to restoring neural cell homeostasis and promoting […]
Taysha administered the first dose of investigational gene therapy drug
These are definitely exciting and hopeful times for Rett community. Taysha Gene Therapies have announced that the first patient was dosed with the investigational gene therapy, TSHA-102, in the REVEAL Adult Study, a clinical trial for females 18 years and older living with Rett syndrome. As things move forward, we´d like to share the letter […]
Replays of Network Support and Research Event
We are delighted that the Rett Syndrome Europe Network Support and Research Event was so successful. Many thanks to our funders The European Joint Programme for Rare Diseases Network Support Scheme and our sponsors Anavex, Taysha, Neurogene and Neuren for making this possible. Also thanks to our country hosts in Turkuye, Hungary, UK, Netherlands, Serbia, […]
What to do with therapy-resistant epilepsy – Katharina’s way
Katharina is now 14 ½ years old and has been epileptic since the age of three. In the beginning, the epileptic seizures manifested themselves at long intervals and were somewhat under control with medication. However, as she grew up, the seizures became more frequent and intense. Katharina also developed therapy resistance to all the medications […]
European Rett Syndrome Congress 2023
The Rett Syndrome Association of France, AFSR are delighted to announce that they are hosting the 2023 European Congress in Marseille on 7th and 8th October 2023. The website is now live with the programme and information about the venue and accommodation. Rett Syndrome Europe will be holding their 2023 General Assembly there during the […]
Latest Press Release from Taysha
11th May 2023 Taysha have shared a press release with news about their gene therapy for Rett syndrome.
ActiveRett!
The team at Telethon Kids in Australia led by Dr Jenny Downs have developed a new online resource to help families and carers support people with Rett syndrome to live more physically active lives. They are inviting family members and carers anywhere in the world to look at the ActiveRett web page and evaluate it […]
Update from Neurogene
Since Neurogene received a lot of questions from the Rett community about the upcoming gene therapy trial they announced earlier this year, they prepared a letter with some answers to the frequently asked questions. Please read the document bellow.
Neurotech International to launch clinical program into use of lead drug NTI164 to treat Rett Syndrome
Clinical-stage biopharmaceutical development company Neurotech International (ASX: NTI) has launched a new clinical program into the use of its daily oral cannabinoid-based drug to potentially treat Rett Syndrome in female patients. The program will comprise a filing for Human Research Ethics Committee (HREC) approval and the commencement of a phase two clinical trial investigating the […]
Taysha Gene Therapy Update
In their press release dated 28th March 2023 Taysha shared important updates to their gene therapy programme for Rett syndrome. Read the full press release here. Taysha has initiated screening of the first potential participant in the Phase 1/2 REVEAL adult study in Canada and is planning to dose the first participant with the investigational […]