Research
Advancing Rett syndrome research across Europe.
RSE works alongside European clinicians, researchers and member associations to accelerate the science that improves outcomes for people with Rett syndrome and their families.
Scientific Advisory Board
Guided by leading European experts
RSE’s Scientific Advisory Board brings together leading European clinicians and researchers — paediatricians, neurologists, geneticists, therapists and care specialists — who help shape our scientific priorities.
They review evidence, contribute to international guidelines on Rett syndrome care, and help RSE focus its work where it can have the greatest impact for families.
Research news
Latest from the lab.
Findings, conference reports and partner updates from our research community across Europe.
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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 full response below.
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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 adverse side effects. They are in discussion with the FDA about moving to the next phase which is dosing more…
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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 Benefit Over Placebo for the Ability to Communicate in Rett Syndrome.
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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 with the higher dose now, and the second paediatric patient enrolled for the higher dose.
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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 have the go ahead to dose the second patient with the higher dose and agreement reached to expand the trial…
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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, motor skills, communication/socialisation and autonomic dysfunction from parent carers and clinicians view points. A further update will be shared later…