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Longitudinal quality of life analysis in a phenylketonuria cohort provided sapropterin dihydrochloride
BACKGROUND: Sapropterin dihydrochloride effectively lowers plasma phenylalanine (Phe) for at least a third of phenylketonuria (PKU) patients, with potential for increased dietary Phe tolerance and decreased medical food requirement. OBJECTIVE: To investigate long-term quality of life (QOL) in patien...
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| Autors principals: | , , , |
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| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
BioMed Central
2013
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| Matèries: | |
| Accés en línia: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3880979/ https://ncbi.nlm.nih.gov/pubmed/24373161 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1477-7525-11-218 |
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