Self-reported treatment-associated symptoms among patients with urea cycle disorders participating in glycerol phenylbutyrate clinical trials

Sandesh C.S. Nagamani; George A. Diaz; William Rhead; Susan A. Berry; Cynthia Le Mons; Uta Lichter-Konecki; James Bartley; Annette Feigenbaum; Andreas Schulze; Nicola Longo; William Berquist; Renata Gallagher; Dennis Bartholomew; Cary O. Harding; Mark S. Korson; Shawn E. McCandless; Wendy Smith; Jerry Vockley; David Kronn; Robert Zori; Stephen Cederbaum; J. Lawrence Merritt; Derek Wong; Dion F. Coakley; Bruce F. Scharschmidt; Klara Dickinson; Miguel Marino; Brendan H. Lee; Masoud Mokhtarani

doi:10.1016/j.ymgme.2015.08.002

Self-reported treatment-associated symptoms among patients with urea cycle disorders participating in glycerol phenylbutyrate clinical trials

Sandesh C.S. Nagamani, George A. Diaz, William Rhead, Susan A. Berry, Cynthia Le Mons, Uta Lichter-Konecki, James Bartley, Annette Feigenbaum, Andreas Schulze, Nicola Longo, William Berquist, Renata Gallagher, Dennis Bartholomew, Cary O. Harding, Mark S. Korson, Shawn E. McCandless, Wendy Smith, Jerry Vockley, David Kronn, Robert ZoriStephen Cederbaum, J. Lawrence Merritt, Derek Wong, Dion F. Coakley, Bruce F. Scharschmidt, Klara Dickinson, Miguel Marino, Brendan H. Lee, Masoud Mokhtarani

Research output: Contribution to journal › Article › peer-review

Original language	English
Pages (from-to)	29-34
Number of pages	6
Journal	Molecular Genetics and Metabolism
Volume	116
Issue number	1-2
DOIs	https://doi.org/10.1016/j.ymgme.2015.08.002
State	Published - Sep 1 2015
Externally published	Yes

ASJC Scopus Subject Areas

Endocrinology, Diabetes and Metabolism
Biochemistry
Molecular Biology
Genetics
Endocrinology

Keywords

Ammonia
Glycerol phenylbutyrate
Health-related quality of life
Patient-reported outcomes
Sodium phenylbutyrate
Treatment-related symptoms

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10.1016/j.ymgme.2015.08.002

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Nagamani, S. C. S., Diaz, G. A., Rhead, W., Berry, S. A., Le Mons, C., Lichter-Konecki, U., Bartley, J., Feigenbaum, A., Schulze, A., Longo, N., Berquist, W., Gallagher, R., Bartholomew, D., Harding, C. O., Korson, M. S., McCandless, S. E., Smith, W., Vockley, J., Kronn, D., ... Mokhtarani, M. (2015). Self-reported treatment-associated symptoms among patients with urea cycle disorders participating in glycerol phenylbutyrate clinical trials. Molecular Genetics and Metabolism, 116(1-2), 29-34. https://doi.org/10.1016/j.ymgme.2015.08.002

Nagamani, SCS, Diaz, GA, Rhead, W, Berry, SA, Le Mons, C, Lichter-Konecki, U, Bartley, J, Feigenbaum, A, Schulze, A, Longo, N, Berquist, W, Gallagher, R, Bartholomew, D, Harding, CO, Korson, MS, McCandless, SE, Smith, W, Vockley, J, Kronn, D, Zori, R, Cederbaum, S, Lawrence Merritt, J, Wong, D, Coakley, DF, Scharschmidt, BF, Dickinson, K, Marino, M, Lee, BH & Mokhtarani, M 2015, 'Self-reported treatment-associated symptoms among patients with urea cycle disorders participating in glycerol phenylbutyrate clinical trials', Molecular Genetics and Metabolism, vol. 116, no. 1-2, pp. 29-34. https://doi.org/10.1016/j.ymgme.2015.08.002

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author = "Nagamani, {Sandesh C.S.} and Diaz, {George A.} and William Rhead and Berry, {Susan A.} and {Le Mons}, Cynthia and Uta Lichter-Konecki and James Bartley and Annette Feigenbaum and Andreas Schulze and Nicola Longo and William Berquist and Renata Gallagher and Dennis Bartholomew and Harding, {Cary O.} and Korson, {Mark S.} and McCandless, {Shawn E.} and Wendy Smith and Jerry Vockley and David Kronn and Robert Zori and Stephen Cederbaum and {Lawrence Merritt}, J. and Derek Wong and Coakley, {Dion F.} and Scharschmidt, {Bruce F.} and Klara Dickinson and Miguel Marino and Lee, {Brendan H.} and Masoud Mokhtarani",

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AU - Nagamani, Sandesh C.S.

AU - Diaz, George A.

AU - Rhead, William

AU - Berry, Susan A.

AU - Le Mons, Cynthia

AU - Lichter-Konecki, Uta

AU - Bartley, James

AU - Feigenbaum, Annette

AU - Schulze, Andreas

AU - Longo, Nicola

AU - Berquist, William

AU - Gallagher, Renata

AU - Bartholomew, Dennis

AU - Harding, Cary O.

AU - Korson, Mark S.

AU - McCandless, Shawn E.

AU - Smith, Wendy

AU - Vockley, Jerry

AU - Kronn, David

AU - Zori, Robert

AU - Cederbaum, Stephen

AU - Lawrence Merritt, J.

AU - Wong, Derek

AU - Coakley, Dion F.

AU - Scharschmidt, Bruce F.

AU - Dickinson, Klara

AU - Marino, Miguel

AU - Lee, Brendan H.

AU - Mokhtarani, Masoud

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KW - Glycerol phenylbutyrate

KW - Health-related quality of life

KW - Patient-reported outcomes

KW - Sodium phenylbutyrate

KW - Treatment-related symptoms

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Self-reported treatment-associated symptoms among patients with urea cycle disorders participating in glycerol phenylbutyrate clinical trials

ASJC Scopus Subject Areas

Keywords

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