Author: Becky Jenner

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 […]

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 […]

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 […]

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 across a number of different symptom areas.