In the report "Spinal Muscular Atrophy and Treatment of SMA in Poland. New quality of life for patients and carers ”, the first data from real clinical observation for pediatric groups are presented. How would you summarize the results of this observation?
First of all, we have a very large group of children whose treatment was successfully summarized. This is a group of 298 patients, of which only six did not complete the first year of treatment. The rest of the group, i.e. 292 children, continued treatment longer. It is a treatment that can be kept safe and effective for a long time. We have not had any case of a child whose treatment had to be discontinued due to intolerance. We also did not have any case of a child who met the criteria of therapy failure. Such criteria include failu...
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