Mike Snape, PhD, chief scientific officer at AMO Pharma explains the different methods currently being used to treat and quell the effects of myotonic dystrophy.
Transcription:
Currently, patients with myotonic dystrophy have non-pharmacological support. They may have aids to walk, for example, and they may have speech therapy. They will then have interventions for their bodily symptoms. I mentioned 25% of them will be treated by a cardiologist because the condition can affect their heart function. Then they will receive a little bit of pharmacological intervention. They may have assistance for issues within their digestive system, constipation, and so on.
They may have treatment to help them with hyperactive behavior or the opposite, sleepiness. They may have behavior if they have myotonia, which is an abnormal contraction of muscles, they can occasionally be given drugs like mexiletine to help with that.
I don’t want to sound dismissive, while they have symptomatic multidisciplinary support, they don’t have anything that treats the underlying condition.
To learn more about this and other musculoskeletal disorders, go to https://checkrare.com/diseases/musculoskeletal-diseases/