Frequently Asked Questions
Q. What is the purpose of SANTA?
We know that the biggest problem for young children with NF1 and their families in practice often relates to learning and social problems. We have done a recent study, which has discovered for the first time that 25% of children with NF1 have autism spectrum disorder and a further 20% of children with NF1 have some partial features of autism. We think that this is an important discovery to explain why some of the social difficulties occur in children with NF1. It suggests new ways of understanding families’ problems and approaching treatment.
In other research, a group of medicines called statins have been shown to improve the functioning of cells and NF1 associated learning problems and behaviour in mice; and to possibly improve brain functioning in older children and adolescents with NF1.
For this reason, we wish to investigate whether the use of statins in young children with NF1 who also have autism will improve their brain functioning and social, communication, and behavioural problems.
Q Who funds the study and who approves it?
The research is organised through the department of Genetic Medicine at the Central Manchester University Hospitals NHS Foundation Trust. The funding comes from the Central Manchester Foundation Trust.
The study is approved by the NHS Central Manchester Ethics Committe Research Department.
Q I would like to find out more about travel arrangements.
We will always cover your travel expenses when you come to visit our research centre which is based in Manchester. Depending on where you live, a taxi service may be provided.
Otherwise, we will reimburse any expenses directly related to the visit.
Q Can I bring my other children to the research centre?
You are more than welcome to bring other siblings. We have designated area for them to play and can arrange for someone to sit with them during your visit. However, we do ask that you let us know in advance if you are bringing anyone else, so that we can make the necessary arrangements.
Q What are the potential benefits of the study?
A study like this gives us valuable general knowledge about children with NF1 and their difficulties in communication and making friendships. We will be able to use this knowledge hopefully to help improve life for children in general with this problem. The study assessments done on all children in the study will give valuable and detailed information about your child’s development and functioning including whether or not your child has an underlying Autistic Spectrum Disorder. This information may be generally useful in accessing and planning the right kind of special help for your child growing up – for instance in school.
Q What if I change my mind and do not want to take part anymore?
You are free to withdraw from the trial at any point and it is perfectly safe to do so. We only ask that you let us know if you are considering this.