Diagnostic and therapeutic recommendations for the treatment of hyperphenylalaninemia in patients 0–4 years of age

Diagnostic and therapeutic recommendations for the treatment of hyperphenylalaninemia in patients 0–4 years of age

Abstract Background Treatment of phenylketonuria (PKU) with sapropterin dihydrochloride in responsive patients from an early age can have many advantages for the patient over dietary restriction alone. Accordingly, approval of sapropterin in the European Union was extended in 2015 to include patient...

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Bibliographic Details
Main Authors: Ania C. Muntau, Marcel du Moulin, Francois Feillet
Format: Article
Language:English
Published: BMC 2018-09-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Infants
Diagnosis
Therapy recommendations
Sapropterin dihydrochloride
Phenylketonuria
BH4
Online Access:http://link.springer.com/article/10.1186/s13023-018-0911-6

Internet

http://link.springer.com/article/10.1186/s13023-018-0911-6

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