Longitudinal quality of life analysis in a phenylketonuria cohort provided sapropterin dihydrochloride

@inproceedings{Douglas2013LongitudinalQO,
  title={Longitudinal quality of life analysis in a phenylketonuria cohort provided sapropterin dihydrochloride},
  author={Teresa Dawn Douglas and Usha Ramakrishnan and Julie A Kable and Rani H. Singh},
  booktitle={Health and quality of life outcomes},
  year={2013}
}
BackgroundSapropterin 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.ObjectiveTo investigate long-term quality of life (QOL) in patients with phenylketonuria (PKU) who took sapropterin (BH4, Kuvan®) for up to one year.Methods37 PKU patients, ages 10–49 years, were asked to complete a PKU-specific self-report QOL questionnaire (QOLQ) at… CONTINUE READING
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