Research Training Fellowship*: Dr Sarah McCarrison
In the UK, around 100 boys are born with Duchenne muscular dystrophy (DMD) each year, a genetic condition that causes progressive muscle weakness and wasting.[1] Children with DMD are also at increased risk of osteoporosis, a condition where bones become weak, fragile, and more prone to fractures. *Dr Sarah McCarrison at the Royal Hospital for Children Glasgow is developing a tool to help predict spine fractures in young people with DMD. Her goal is to enable more personalised care, with earlier treatment to help prevent fractures in those most at risk, while sparing others from the potential side effects of unnecessary medications.
How are children’s lives affected now?
Young people with DMD are at higher risk of osteoporosis, a condition where bones weaken – and as a result, can fracture more easily.
“Fractures of the backbones in the spine – called vertebrae – are very common in boys with DMD,” says Dr McCarrison. “Unlike a broken arm or leg, a spine fracture can be less obvious, but it can still be painful and may lead to long-term back problems that can further impact a child’s quality of life.”
Currently, if a spine fracture is diagnosed, doctors prescribe bone-strengthening medications to help prevent further fractures. However, these treatments may also cause serious side effects, so they are only started once a fracture has already occurred.
Starting bone-strengthening medications sooner may help prevent spine fractures from happening in the first place – but currently, doctors have no reliable way of identifying which boys are most at risk and could benefit from early treatment.
How could this research help?
“We’re aiming to identify factors that can predict spine fractures in young people with DMD – and to develop a risk assessment tool to support more personalised care,” says Dr McCarrison.
The researchers will analyse clinical data – including medical records and spinal scans – from up to 250 boys with DMD, collected as part of their routine treatment at centres across the UK.
“Using statistical and advanced computer-based methods, we will develop a risk score, based on factors we identify as early indicators of fractures,” says Dr McCarrison. “We will then test the accuracy of this tool by following a separate group of 50 boys with DMD over a two-year period.”
The team will also hold focus groups with doctors who care for children with DMD, to explore how this new tool could be integrated into clinical practice.
We hope this will ultimately lead to a more tailored approach for managing fragile bones in boys with DMD – helping to ensure that bone-strengthening medications are offered earlier to those most at risk, while sparing others from unnecessary treatment.
Research table
Project details
| Project Leader | Dr Sarah McCarrison, BSc (MedSci) MBChB MRCPCH PgCert |
| Location | Department of Paediatric Endocrinology, Royal Hospital for Children Glasgow |
| Project Team |
Dr Jarod Wong, BSc(Med)/MBBS MRCPCH DMedSc
Professor Konstatinos Gerasimidis, PG Dip Med PhD Dr Nicola Crabtree, PhD Dr Claire Wood, PhD MBChCB BMedSci MRCPCH |
| Other Locations |
Department of Human Nutrition, School of Medicine, Dentistry and Nursing, University of Glasgow
Department of Paediatric Endocrinology and Diabetes, Birmingham Women’s and Children’s Hospital Department of Paediatric Endocrinlogy, Translational and Clinical Research Institute, Newcastle University |
| Grant Awarded | |
| Grant Amount | £297,569 |
| Duration | 36 months |
| Grant Code (GN number) | GN3095 |
References
- NHS website, Muscular Dystrophy: https://www.nhs.uk/conditions/muscular-dystrophy/ [website accessed 30 April 2025]
*Research Training Fellowships:
Each year, Action Medical Research awards these prestigious grants to help the brightest and best doctors and scientists develop their career in medical research.
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