Dr Ros Quinlivan at University College London (UCL) is aiming to drive the development of the adult North Star Network. This project will establish a set of standardised outcome measures that will help to improve overall care for adults with Duchenne muscular dystrophy.
The Adult NorthStar Network has been established across 17 neuromuscular centres in the UK. The network co-ordinator, Dr Laura Nastasi, has worked closely with NorthStar clinicians to establish a set of outcome measures that are meaningful for adults with Duchenne muscular dystrophy. The data entry forms to capture this information have been designed and added to the existing NorthStar database.
The regulatory approvals that are necessary for the adult part of the database have been obtained. This includes ethical approval, which ensures that the research will be properly conducted and that patients are protected.
The team have been working closely with the Adult NorthStar Network to develop a standards of care document for adults with Duchenne living in the UK. This is still in progress and we will keep you updated on their publication.
What are the aims of the project?
This PhD studentship will drive the development of the adult North Star Network. For children, the North Star Network has established regular assessments that monitor a range of things, including walking ability and muscle strength. This helps better understand the progression of the condition (referred to as natural history), helps improve the care that is offered to children and provides outcome measures for clinical trials. However, for adults, similar measures are not appropriate and it is important to define better outcome measures for this group of people.
This PhD project will establish a set of standardised outcome measures that are appropriate for adults with Duchenne muscular dystrophy. It will train clinics across the UK in how to collect these measurements and upload this data to an electronic database. This will provide important natural history information on up to 500 adults living with Duchenne muscular dystrophy in the UK.
Why is this research important?
Thanks to improvements in clinical care, adults with Duchenne muscular dystrophy are a relatively new group of patients attending neuromuscular clinics. However previous research has shown that their access to appropriate multi-disciplinary care in the UK is poor compared with some other European countries. This PhD studentship hopes to address this problem by developing standardised outcome measures that will improve overall care.
Using a set of standardised outcome measures is essential for collecting good quality data in clinical trials. The findings of this project will therefore help with the designing of future trials for adults with Duchenne muscular dystrophy.
How will the outcomes of this research benefit patients?
This PhD project will drive the adult North Star Network, engage healthcare professionals and ultimately improve the standard of care for adults with Duchenne muscular dystrophy. Comprehensive natural history data and the development of new outcome measures will also help pave the way for more clinical trials.
How might this research impact on other neuromuscular conditions?
This project will identify outcome measures that could be useful for many different neuromuscular conditions, for example measurements that assess bowel and kidney function.
Project leader: Dr Ros Quinlivan
Institute: UCL (Queens Square Centre for Neuromuscular Diseases)
Condition(s): Duchenne muscular dystrophy
Duration: four years, starting 2017
Total cost: £180,119
Official title: A natural history study of a cohort of adults with Duchenne Muscular Dystrophy living in the UK
Find out more from Dr Quinlivan in this video
Read about our other Duchenne muscular dystrophy research projects
Read our research news stories on Duchenne muscular dystrophy
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