Regarding the CHMP Opinion on Trofinetide The European Medicines Agency (EMA) has announced that its Committee for Medicinal Products for Human Use (CHMP) has recommended not granting marketing authorisation for Trofinetide for the treatment of Rett syndrome in Europe. According to EMA, the CHMP concluded that the treatment effects observed after 12 weeks were not sufficient […]
Registration is now open for the International Rett Syndrome Conference in Istanbul! A unique gathering for doctors, scientists, families, and therapists! Be a part of this important event and stay up to date with the latest scientific developments in the Rett syndrome field! Register here now! The International Rett Syndrome Conference 2025 is now open […]
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.
To mark Rare Disease Day 2025 we are sharing the amazing adventures of Selena – an very inspiring young lady with Rett syndrome and her equally wonderful family!
Research updates, events and family focus stories plus an opportunity to hear from Tom Pulles and Stephanie Kim from Acadia on 4th March 2025, 7pm CET – you will need to register first to join the webinar.
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 […]
Many thanks to Usree and Kalika for sharing this amazing account of their trip across several European countries and India during the monsoon season! It is a such an interesting and inspirational reading but also full of really helpful tips of what to take and things to consider when travelling with someone with Rett syndrome. […]
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.
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.
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 […]
