The average cost of idursulfase treatment in Italy exceeds 300.000 €/year for a 30 kg patient and proportionally increases with patients’ weight; compared to this, the costs of treatment monitoring appear almost insignificant and necessary to assess drug efficacy in every single patient.
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Clinical efficacy of enzyme replacement therapy in paediatric Hunter patients, an independent study of 3.5 years.
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The sentences
Overall, although a positive trend could be observed, the paucity of data did not allow to draw any conclusions about the efficacy of the therapy on this performance.
Similarly, weight falls within the reference standards up to about 15 years of age, showing later on a negative trend [ 29 , 30 ].