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
Taysha share more information on Part A and Part B of their gene therapy trial
More details on the changes seen so far in the first cohort of individuals who received either the high or low dose, ages 6-21 years. No serious adverse events seen at either dose. Read the …
Latest Update From Taysha
We are delighted to share the very positive update from Taysha on their gene therapy programme for Rett syndrome. Ten participants have now received the therapy which has generally been well tolerated with no serious …
Acadia confirm submission to the EMA for approval of trofinetide (known as Daybue in US and Canada).
You can read the full press release here . They expect it will be 10-14 months before a decision is known. You might also like to read this plain language summary Trofinetide Treatment Demonstrates a …
Latest update from Taysha …
You can read their latest letter to the Rett community here. All patients dosed are generally tolerating the therapy well with no serious adverse side effects. Third patient in the adult cohort has been dosed …
Latest update from Taysha
We are delighted to be able to share the latest update from the Taysha gene therapy programme. Highlights include the first person who received the higher dose has had no serious adverse side effects, they …
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 …