In RSE we understand that research is essential for overcoming Rett Syndrome one day. We also know that it is one of the main topics of interest for families and everyone who faces Rett in their life. Therefor, RSE is taking new actions to keep you up to date with all relevant information regarding research in Rett Syndrome.

Scientific Advisory Board

Rett Syndrome Europe are delighted to announce we now have a Scientific Advisory Board!

Scientific Advisory Board members are:

Gill Townend – Chair of SAB
Aglaia Vignoli, Ana Abdala, Anne-Marie Bisgaard, Bernd Wilken, Bojana Milanov, Danijela Szili, Helena Wandin, Jean-Christophe Roux, Meir Lotan, Michelle Stahlhut, Peter Marschik, Sonia Guil.

Clinical Trials

Information on clinical trials is constantly changing, but we will be posting latest updates in our research blog to keep you up to date as much as possible on the latest advancements. However, for the full list of complete information on clinical trials in Rett Syndrome, please visit the link below:

Research news

Latest Update from Taysha
In the latest update published today we learn that there have been no serious adverse effects from the treatment in both the adult and paediatric trials so far. Both groups are reporting improvements in seizures, …
Latest update from Taysha
Taysha today shared updates on their REVEAL Adolescent and Adult Study. Here is the letter to the community. Also read their press release though as there is much more detail there about the improvements seen …
Update from Neurogene Clinical Trial
Good news from Neurogene as they progress their gene therapy trial to the next stages. You can read their letter to the community here. neurogene-our-letter-to-rett-syndrome-community-march-2024Download
Latest Update on Taysha Gene Therapy
Summary The REVEAL Adolescent & Adult Study (females age 12+) will expand into the United States (U.S.) following submission of an investigational new drug application to the Food and Drug Administration (FDA). The REVEAL Adolescent …
A plain language summary of results from the LAVENDER study: trofinetide treatment for Rett syndrome
Many thanks to Future Rare Diseases for sharing this publication with us. You can read the full paper here.
Study about Males with Rett Syndrome
Dr Tim Benke, Medical Director of the Rett Center of Excellence at Colorado Children’s Hospital is the lead Principal Investigator in a study that is seeking to understand the lived experience of Males with Rett …
Jan 24 Update from Taysha
Official press release from Taysha giving more detail of their REVEAL paediatric gene therapy trial with sites in the US and UK. Taysha-REVEAL-Pediatric-Study-Announcement-Jan-2024-1Download
Taysha Gene Therapy Trial Update
Taysha announce details of their REVEAL paediatric study in the US which is now recruiting patients. Taysha-REVEAL-Pediatric-Study-US-Trial-Enrollment-LetterDownload
Taysha expand age group in gene therapy trial in Canada to 12+ years.
The latest press release from Taysha announces that they have got the go-ahead to include adolescents age 12 years and over in their trial. The third patient to be dosed is expected to be in …
Latest update from Taysha gene therapy
Taysha today shared the latest update on their gene therapy programme with the second person now having received the therapy and a third planned for later this year. You can read their announcement and their …
Latest Update From Taysha Gene Therapy Programme
News on the first patient dosed with their gene therapy and next steps in respect of expanding the trials… Taysha-Rett-Community-Letter-August-14-2023Download
Latest Update From Taysha Gene Therapy Programme
Taysha have shared the news today that they have been given the all clear to continue with their plans to dose the second adult with Rett syndrome in their gene therapy trial in Canada. You …
The Burden of Illness Study in Rett Syndrome
Following our Network Support and Research Event we would like to share a video of Walter Kaufmann presentation "Burden of Illness in Rett Syndrome: Initial Evaluation of a Disorder-Specific Caregiver Survey". In this talk, Walter …
Update from Anavex Life Sciences clinical trial
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 …
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 …
Latest Press Release from Taysha
11th May 2023 Taysha have shared a press release with news about their gene therapy for Rett syndrome. Taysha has completed screening and scheduled dosing for the first potential participant in the Phase 1/2 REVEAL …
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 …
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 …
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 …
Network Support and Research Event Registration – Replays Available!
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, …

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