Sapropterin dihydrochloride for phenylketonuria.

ABSTRACT:

Background

Phenylketonuria results from a deficiency of the enzyme phenylalanine hydroxylase. Dietary restriction of phenylalanine keeps blood phenylalanine concentration low. Most natural foods are excluded from diet and supplements are used to supply other nutrients. Recent publications report a decrease in blood phenylalanine concentration in some patients treated with sapropterin dihydrochloride. We examined the evidence for the use of sapropterin dihydrochloride to treat phenylketonuria. This is an update of a previously published Cochrane Review. 

Objectives

To assess the safety and efficacy of sapropterin dihydrochloride in lowering blood phenylalanine concentration in people with phenylketonuria.

Search methods

We identified relevant trials from the Group's In

SUBMITTER: Somaraju UR 

PROVIDER: S-EPMC6769157 | biostudies-literature | 2015 Mar

REPOSITORIES: biostudies-literature