Myotonic dystrophy

To participate in this study, contact: Alastair Corbett

Research site: Concord Hospital, Sydney

Current situation

Animal models and tissue culture systems have been developed to assess the results of potential therapies.

Future potential therapy

There is the likelihood of human trials within 5 years.

There are a number of collaborative opportunities in this area:

• Clinical markers of disease progression or biomarkers to be used for future therapeutic trials
• Longitudinal studies – registries
• Management of cardiac complications
• Cognitive impairment
• Management of fatigue and sleep disturbance
• Susceptibility to infection