Self-Reported Treatment-Associated Symptoms among Patients with Urea Cycle Disorders Participating in Glycerol Phenylbutyrate Clinical Trials

BACKGROUND: Health care outcomes have been increasingly assessed through health-related quality of life (HRQoL) measures. While the introduction of nitrogen-scavenging medications has improved survival in patients with urea cycle disorders (UCDs), they are often associated with side effects that may...

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Vydáno v:Mol Genet Metab
Hlavní autoři: Nagamani, Sandesh C. S., Diaz, George A., Rhead, William, Berry, Susan A., Le Mons, Cynthia, Lichter-Konecki, Uta, Bartley, James, Feigenbaum, Annette, Schulze, Andreas, Longo, Nicola, Berquist, William, Gallagher, Renata, Bartholomew, Dennis, Harding, Cary O., Korson, Mark S., McCandless, Shawn E., Smith, Wendy, Vockley, Jerry, Kronn, David, Zori, Robert, Cederbaum, Stephen, Merritt, J. Lawrence, Wong, Derek, Coakley, Dion F., Scharschmidt, Bruce F., Dickinson, Klara, Marino, Miguel, Lee, Brendan H., Mokhtarani, Masoud
Médium: Artigo
Jazyk:Inglês
Vydáno: 2015
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On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4804346/
https://ncbi.nlm.nih.gov/pubmed/26296711
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2015.08.002
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