Characterising pre-clinical models and testing gene therapy for X-linked spinal muscular atrophy
Developing blood tests for improved diagnosis of muscular dystrophy and measuring the progression of neuromuscular conditions
Using MRI to monitor the progression of Duchenne muscular dystrophy
Using antibiotics to treat symptoms of myotonic dystrophy type 1
A pilot study to establish a new tool to investigate motor skills in SMA mouse models
Assessing a non-invasive approach to detect myopathy in children
Identification of Charcot-Marie-Tooth disease therapies through drug repurposing
