The study of this survey was an interesting exercise because we as an ERN EURO NMD submitted the survey to more than 1500 patients with any kind of neuromuscular disease. And what we have seen is that considering all the most bothersome symptoms that patients may experience during the disease course, there are four or five common symptoms that are present across all different neuromuscular diseases...
The study of this survey was an interesting exercise because we as an ERN EURO NMD submitted the survey to more than 1500 patients with any kind of neuromuscular disease. And what we have seen is that considering all the most bothersome symptoms that patients may experience during the disease course, there are four or five common symptoms that are present across all different neuromuscular diseases. This is an important starting point because we now know that across the neuromuscular community we have selected symptoms that are almost always present in any kind of neuromuscular disorder. So the idea is now to build a specific problem, a specific patient-reported outcome measure that is going to focus on these specific symptoms that can be used to monitor patients longitudinally and also in response to clinical trials or rehab protocols and this can be applied theoretically to all neuromuscular patients.
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