TREATMENT FOR RETT SYNDROME: WHAT DOES IT MEAN?

With a number of potential treatments for Rett in clinical trials, one having been approved and more projected to move to clinical trials, it's important for families to understand what these treatments are, how they work and what symptoms might improve as a result of trying the treatment. The word 'treatment' means medical care given to a patient for illness or injury.

This covers lots of different kinds of treatments It could cover medications or drugs given as tablets, liquid medicines or injections. It could also cover gene therapies, transplants, operations and surgeries as well as different kinds of therapies amongst other things

At this time, in Rett Syndrome, we are mainly seeing two different kinds of treatments moving into clinical trials; those which are medicines aiming to treat some of the symptoms of Rett Syndrome and those which are more invasive treatments, such as gene therapies, which are aiming to treat the underlying cause of the disease

Drug treatments include medicines such as:

Anavex 2-73 - currently being trialled in children Adult study is complete

Trofinetide / Daybue - which has recently been approved by the FDA.

More invasive treatments aiming to treat the underlying cause of Rett include:

Taysha Gene Therapies TSHA-102 - currently being trialled in adults in Canada, projected to apply to hold a UK clinical trial for children later this year.

Neurogene NGN-401 - which has recently received the green light for a children's trial to start in the USA

The most common question we receive at Reverse Rett is...

When will treatments for Rett Syndrome be available for my child?

The nature of research and clinical trials makes it difficult to predict exactly how long the development or trialling of a given treatment will take.

Here are some upcoming milestones to look for:

June / July 2023

Taysha Gene Therapies update re top line results from adult trial of TSHA-102

July / August 2023

Neurogene's US clinical trial for NGN 401 in children going up on clinicaltrials.gov website

June / August 2023

Taysha Gene Therapies update re proposed application to UK regulator for TSHA-102 in children

You can find information about all clinical trials worldwide on the clinicaltrials.gov website and filter your search for Rett Syndrome and UK