Measuring Burden of Illness in Phenylketonuria (PKU): Development of the PKU Symptom Severity and Impacts Scale as a Robust Patient-Reported Outcome

por Quinn, Jennifer, Georgiadis, Alexandros, Lewis, Hannah B., Jurecki, Elaina
Publicado 2021

Development of the US English version of the phenylketonuria – quality of life (PKU-QOL) questionnaire

por Jurecki, Elaina, Cunningham, Amy, Birardi, Vanessa, Gagol, Grégory, Acquadro, Catherine
Publicado 2017

Sapropterin Dihydrochloride Mixed With Common Foods and Beverages

por Jurecki, Elaina R., Cunningham, Amy, Mahoney, John J., Tingley, Douglas, Chung, Stanley, James, Neil, Cohen-Pfeffer, Jessica L.
Publicado 2014

Long-term comparative effectiveness of pegvaliase versus standard of care comparators in adults with phenylketonuria

por Zori, Roberto, Ahring, Kirsten, Burton, Barbara, Pastores, Gregory M., Rutsch, Frank, Jha, Ashok, Jurecki, Elaina, Rowell, Richard, Harding, Cary
Publicado 2019

Newborn screening 50 years later: access issues faced by adults with PKU

por Berry, Susan A., Brown, Christine, Grant, Mitzie, Greene, Carol L., Jurecki, Elaina, Koch, Jean, Moseley, Kathryn, Suter, Ruth, van Calcar, Sandra C., Wiles, Judy, Cederbaum, Stephen
Publicado 2013

Improved attention linked to sustained phenylalanine reduction in adults with early‐treated phenylketonuria

por Bilder, Deborah A., Arnold, Georgianne L., Dimmock, David, Grant, Mitzie L., Janzen, Darren, Longo, Nicola, Nguyen‐Driver, Mina, Jurecki, Elaina, Merilainen, Markus, Amato, Gianni, Waisbren, Susan
Publicado 2021

A non-interventional observational study to identify and validate clinical outcome assessments for adults with phenylketonuria for use in clinical trials

por Burton, Barbara K., Skalicky, Anne, Baerwald, Christoph, Bilder, Deborah A., Harding, Cary O., Ilan, Aaron B., Jurecki, Elaina, Longo, Nicola, Madden, David T., Sivri, H. Serap, Wilcox, Gisela, Thomas, Janet, Delaney, Kathleen
Publicado 2021

Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome

por Hendriksz, Christian J., Berger, Kenneth I., Parini, Rossella, AlSayed, Moeenaldeen D., Raiman, Julian, Giugliani, Roberto, Mitchell, John J., Burton, Barbara K., Guelbert, Norberto, Stewart, Fiona, Hughes, Derralynn A., Matousek, Robert, Jurecki, Elaina, Decker, Celeste, Harmatz, Paul R.
Publicado 2016