ASD in children with MPS III
Research type
Research Study
Full title
The symptomology of Autistic Spectrum Disorder in children with Mucopolysaccharide Disease III (MPS III)
IRAS ID
192590
Contact name
Claire Wolfenden
Contact email
Sponsor organisation
University of Manchester
Duration of Study in the UK
1 years, 1 months, 3 days
Research summary
Mucopolysaccharide disease type III (MPS III) is a rare genetic disorder occurring in around 1 in 89,000 births and which is severely life-limiting with mortality in the second and third decades. At present, there are no treatments for MPS III although a number of possible treatments are currently being trailed at the Royal Manchester Children’s Hospital. There are four main sub-types of MPS III (A, B, C & D), A being the most common and D rare. Children with all sub-types of MPS III can present with challenging behaviour and recent research explored potential mechanisms. Previous research has identified that children with MPS III are at risk of co-morbid Autistic Spectrum Disorders (ASD), although diagnoses often overlap in the case of genetic disorders. Therefore this study will look at whether children with MPS III have symptoms of ASD rather than a diagnosis of ASD and examine whether there are any differences related on the age of the child and/or the sub-type of MPS III It is hoped that this will help clinicians to identify appropriate ways of supporting these children and their families and ultimately improve their quality of life.
This project will recruit parents/carers of children with MPS III from a hospital clinic and through the support of the MPS society. They will be asked to complete questionnaires that examine whether their children have behaviours typical of a child with ASD. The child’s age and sub-type of MPS III will also be noted to compare how symptoms of ASD differ across age and sub-type of MPS III.REC name
East Midlands - Leicester Central Research Ethics Committee
REC reference
16/EM/0110
Date of REC Opinion
8 Apr 2016
REC opinion
Further Information Favourable Opinion