Investigators: Andrew Wilson, Graham Hall, Jenny Downs, Jenny Lam, Adelaide Withers, Grace Pettigrew
Neuromuscular disorders affect the muscles used to breathe and can cause significant breathing problems. Problems with breathing often begin during sleep, but it can be difficult to determine when these problems start. Measuring lung function helps monitor disease progression and whether treatments are effective, but many of these lung function tests are difficult for children with neuromuscular disorders to perform.
This study aims to determine whether a combination of lung function tests, motor function tests, sleep measures and quality of life questionnaires are better than conventional tests at monitoring breathing difficulties, and if they can predict worsening disease in children with neuromuscular disease. We hope that through earlier diagnosis and treatment of muscle weakness during sleep, we can prevent future lung failure in children with neuromuscular disorders.
This study is funded by Muscular Dystrophy WA, Duchenne Parent Project and the National Health and Medical Research Council.
External collaborators:
- Hayley Lethlean (Muscular Dystrophy WA)
- Peter Rowe (Perth Children's Hospital)
- Carlos Milla, Mymy Buu, Diana Naranjo, John Day, Tian Lu (Stanford University)