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Developing clinical predictors of disease progression in children with neuromuscular disorders to prevent future respiratory failure

Investigators: Graham Hall, Adelaide Withers, Andrew Wilson, Grace Pettigrew, Jenny Lam

External collaborators: Peter Rowe (Princess Margaret Hospital for Children), Hayley Lethlean (Muscular Dystrophy WA), Carlos Milla (Stanford University)

Partners: Muscular Dystrophy Association of Western Australia, Stichting Duchenne Parent Project Netherlands

This study aims to identify problems that children with neuromuscular conditions experience with breathing during sleep, due to muscle weakness. Participants will undergo various tests to see whether we can diagnose this problem early, rather than relying on the current method of simply asking about symptoms. We hope that through earlier diagnosis and treatment of muscle weakness during sleep, we can prevent future lung failure. Those involved in the research study will have data collected over a 12-month period via lung function tests, motor function tests and questionnaires to be completed during regular clinical appointments.