Interested Families

Who can participate?

We are interested in hearing from families with children diagnosed with NF1 and aged from 5 and up to 9 years old. They do not need to have a diagnosis of Austic Spectrum Disorder in order to participate.

What is SANTA?

SimvaStatin in Neurofibromatosis Type 1- Autism is a research study looking at the use of the drug Simvastatin in children with NF1 and Autism Spectrum Disorders (ASD).

Our recent studies have shown us that about 25% of children with NF1 have Autism Spectrum Disorder. Currently there are no available treatments for these difficulties. We have reason to believe that a medicine called Simvastatin may improve some of these difficulties, but this has not been tested yet. We want to know if Simvastatin is well tolerated and whether it can help children with NF1 and autism. If this study is successful then it could possibly open up an important new avenue of therapy for children who suffer from NF1 as well as autism.

What are Autism Spectrum Disorders?

ASD is a condition which roughly affects 1 in 100 people. It affects communication and how someone relates to other people. It also affects how people make sense of the world around them. It is a ‘spectrum’ condition which means that difficulties vary from child to child from mild to severe. Children with ASD often struggle academically; find it difficult to make friends and ‘fit-in’.

For more information visit: National Autistic Society website

Are there any benefits?

A study like this will tell us more about children with NF1 and their difficulties in communication and making friendships. The study may help to indicate whether your child may have an underlying Autistic Spectrum Disorder. Understanding these difficulties better may allow you to access educational and other services suitable to meet your child’s needs. We will also do some genetic testing and brain scan as part of this study and share the result with you if you wish.

Will others know we are taking part?

No. Everything that you and your child will tell us will be kept confidential, which means that only the people in our research team will know.