Newborn Screening and Treatment of Phenylketonuria: Projected Health Outcomes and Cost-Effectiveness

• Główni autorzy: Huey-Fen Chen (Autor), Angela M. Rose (Autor), Susan Waisbren (Autor), Ayesha Ahmad (Autor), Lisa A. Prosser (Autor)
• Format: Książka
• Wydane: MDPI AG, 2021-05-01T00:00:00Z.
• Hasła przedmiotowe: article
• Dostęp online: Connect to this object online.

The objective of this study was to evaluate the cost-effectiveness of newborn screening and treatment for phenylketonuria (PKU) in the context of new data on adherence to recommended diet treatment and a newly available drug treatment (sapropterin dihydrochloride). A computer simulation model was de...