Understanding The Cause Of Muscle Weakness In Nemaline Myopathy (NM) – Moving Towards The Development Of Targeted Treatments
Funder
National Health and Medical Research Council
Funding Amount
$408,768.00
Summary
Congenital myopathy patients have unremitting, life-long muscle weakness that severely affects their quality of life and ability to perform normal daily activities. Currently no effective therapies exist for these conditions, largely due to our limited understanding of the mechanisms leading to muscle weakness. This ECF aims to determine the cause of weakness and test two therapies which have shown promise for other conditions and can be translated into clinical use for myopathies if effective.