I think that the one of the most important aspects is to have the possibility to intercept what we call the natural history of the disease. We know that the neuromuscular disorders are often very progressive diseases in such a way the clinical picture is going to increase the patient burden and also the burden of the family within the patient’s lives. So also the caregiver problems are very important...
I think that the one of the most important aspects is to have the possibility to intercept what we call the natural history of the disease. We know that the neuromuscular disorders are often very progressive diseases in such a way the clinical picture is going to increase the patient burden and also the burden of the family within the patient’s lives. So also the caregiver problems are very important.
So with a new strategy, see neuromuscular rehabilitation, we have the target to, for these problems and the very important element in these to suggest, to propose to the patient and the family, some practical solutions to be used in everyday life. These to be combined with the more experimental, conventional therapies. So pharmacological therapies that in the last year has become to be ready to treat some very often devastating neuromuscular diseases like Duchenne muscular dystrophy, amyotrophic lateral sclerosis or spinal muscular atrophy.
A timely diagnosis is fundamental. Now they, in at least 50% of the situations, I mean that a precocious diagnosis is important to when you have, you have got an effective treatment or otherwise the importance to do that is in some way limited for that. In doing that, we now have, especially for genetic disorders, but also for all other types of neuromuscular disease, we think about neuro-immunological muscle disease or acquired muscle disease, a number of laboratory possibilities to make a precise diagnosis from a for instance, genetic point of view and DNA analysis by molecular biomarkers. This is important, especially if we think to the so-called personalized medicine. Now we have to think to make choice over very individual characteristics of a treatment also in the more modern drugs.
