Alternative Therapies for PKU
J. inborn errors metab. screen
;
5: e160037, 2017.
Article
Dans Anglais
|
LILACS-Express
| LILACS
| ID: biblio-1090923
ABSTRACT
Abstract The phenylalanine (PHE)-restricted diet has improved in quality and diversity over time and has proven to be effective in all patients. Nevertheless, this treatment imposes a heavy social and economic burden to patient and family and impacts quality of life. Sustained adherence to PHE restriction is difficult to maintain. Moreover, even patients with phenylketonuria (PKU) with normal intelligence quotient (IQ) have lower IQ than matched individuals without PKU and can have deficits in multiple other aspects of neuropsychological function, including cognitive and executive function, working memory. They can also have behavior problems, depression, and low self-esteem. In recent years, alternative treatments for PKU have been developed and their use has been indicated for some patients who are candidates for options besides traditional treatment. Sapropterindihydrochloride, large neutral amino acids, and glycomacropeptide are alternative treatment options in use for selected patients. The aim of this article is to review the current knowledge of these new approaches to PKU treatment.
Texte intégral:
Disponible
Indice:
LILAS (Amériques)
langue:
Anglais
Texte intégral:
J. inborn errors metab. screen
Thème du journal:
Medicina Cl¡nica
/
Patologia
Année:
2017
Type:
Article
Pays d'affiliation:
Argentine
Institution/Pays d'affiliation:
Hospital de Niños R/AR
/
Hospital de Niños de La Plata/AR
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