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Sapropterin: A New Therapeutic Agent for Phenylketonuria
Annals of Pharmacotherapy, 08/05/09
Hegge KA et al. – Sapropterin offers a promising therapeutic option for select individuals with BH4–responsive PKU, although long–term data are limited evaluating its safety and efficacy in traditional clinical practice settings. When considering sapropterin therapy, clinicians must consider factors such as cost and patient adherence to drug therapy and/or diet.
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