You are the co-author of the report "Spinal Muscular Atrophy and SMA Treatment in Poland. New quality of life for patients and carers ”. What are the conclusions of this report?
The most important conclusion from the report on the treatment of patients with spinal muscular atrophy in Poland is that during these few years we have managed to extend the treatment to a very large population of patients of all ages, from children to adults, including presymptomatic patients. What is our greatest joy today is that we see the effectiveness of this therapy in all patients. Not a single patient was excluded from the program due to loss of efficacy. This is a very important argument for treating all patients and seeing their next success...
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