%0 Journal Article
%T A NEW THERAPY IN PHENYLKETONURIA: PEGVALIASE
%A B¨¹£¿ra Erdal
%J -
%D 2018
%X Phenylketonuria (PKU) is an autosomal recessive disease caused by deficiency in phenylalanine hydroxylase enzyme. The cornerstone of PKU therapy is dietary therapy with low phenylalanine (Phe) content. Although Phe restricted diet therapy prevents severe cognitive disorders, the dietary adherence problems experienced for both themselves and their families are increasing especially in adolescence and adulthood. Therefore, new alternative methods to dietary therapy or to reduce restrictions in the diet are being investi-gated. Pegvaliase, approved by the U.S. Food and Drug Administration in May 2018, has been a promising enzyme therapy for adults with PKU. Pegvaliase, given as enzyme substitution, converts excess Phe in the blood into low levels of ammonia and non-toxic trans-cinnamic acid. In clinical studies, it has been shown that Pegvaliase is effective in decreasing the blood Phe level in adults with PKU and in-crease the amount of protein and Phe provided by natural protein sources in their diets and decrease the amount of protein provided by medical foods. Some patients treated with Pegvaliase applied an unrestricted diet, while others continued to use a restricted diet from the Phe. In conclu-sion, Pegvaliase therapy is a very new method, and further-more research is needed to explain its relationship with diet therapy
%K Fenilketon¨¹ri
%K Pegvaliase
%K PEG-PAL
%K Yeni tedavi
%K Yeti£¿kin
%U http://dergipark.org.tr/erusaglik/issue/42334/509608