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Sapropterin therapy has been used to manage phenylketonuria (PKU), a genetic disorder characterized by a deficiency of the enzyme phenylalanine hydroxylase, which leads to an accumulation of phenylalanine in the body, causing various neurological symptoms [1]. Sapropterin, a synthetic form of tetrahydrobiopterin, acts as a cofactor for phenylalanine hydroxylase, thereby reducing phenylalanine levels [1].

However, it is essential to note that sapropterin therapy does not ensure continuous symptom relief for all patients with PKU [1]. The effectiveness of sapropterin therapy varies among individuals, and it is most effective in patients with mild to moderate PKU who have some residual enzyme activity [1]. Furthermore, sapropterin therapy does not eliminate the need for dietary restrictions and regular monitoring of phenylalanine levels [1].

Moreover, sapropterin is not a cure for PKU, and discontinuing the therapy may result in a rapid increase in phenylalanine levels and the recurrence of symptoms [1]. Therefore, patients with PKU require lifelong management, including dietary restrictions, regular monitoring of phenylalanine levels, and possibly sapropterin therapy [1].

In summary, sapropterin therapy can help manage PKU symptoms in some patients, but it does not ensure continuous symptom relief for all patients [1]. The effectiveness of sapropterin therapy varies among individuals, and patients with PKU require lifelong management.

Sources:
[1] DrugPatentWatch.com. (n.d.). Sapropterin. Retrieved from <https://www.drugpatentwatch.com/drugs/sapropterin>.