Clinical experience with glycerol phenylbutyrate in 20 patients with urea cycle disorders at a UK paediatric centre

Autor: Mildrid Yeo, Preeya Rehsi, Megan Dorman, Stephanie Grunewald, Julien Baruteau, Anupam Chakrapani, Emma Footitt, Helen Prunty, Melanie McSweeney

Publikováno v: JIMD Reports, Vol 64, Iss 5, Pp 317-326 (2023)

Abstract In urea cycle disorders (UCDs) ammonia scavenger drugs, usually sodium‐based, have been the mainstay of treatment. Increasingly, glycerol phenylbutyrate (GPB, Ravicti®) is being used but scant real‐world data exist regarding clinical ou

Externí odkaz: https://doaj.org/article/cb4630541705478ab681b28299176d20

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BLOOD AMMONIA AND GLUTAMINE AS PREDICTORS OF HYPERAMMONEMIC CRISES IN UREA CYCLE DISORDER PATIENTS

Autor: Lee, Brendan, Diaz, George A, Rhead, William, Lichter-Konecki, Uta, Feigenbaum, Annette, Berry, Susan A, Le Mons, Cindy, Bartley, James A, Longo, Nicola, Nagamani, Sandesh C, Berquist, William, Gallagher, Renata, Bartholomew, Dennis, Harding, Cary O, Korson, Mark S, McCandless, Shawn E, Smith, Wendy, Cederbaum, Stephen, Wong, Derek, Merritt, J Lawrence, Schulze, Andreas, Vockley, Jerry, Kronn, David, Zori, Roberto, Summar, Marshall, Milikien, Douglas A, Marino, Miguel, Coakley, Dion F, Mokhtarani, Masoud, UCD Consortium, Scharschmidt, Bruce F

Publikováno v: Genetics in medicine : official journal of the American College of Medical Genetics
Genetics in medicine : official journal of the American College of Medical Genetics, vol 17, iss 7

PurposeThe aim of this study was to examine predictors of ammonia exposure and hyperammonemic crises in patients with urea cycle disorders.MethodsThe relationships between fasting ammonia, daily ammonia exposure, and hyperammonemic crises were analyz

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::cc91f49be74bc5022b22e4dc57c48988
http://europepmc.org/articles/PMC4465427

Evaluación positiva de medicamentos: septiembre, octubre y noviembre 2015

Autor: Aparicio Hernández,R., García Luque,A., Gonzalo Salado,M.ªH., Cabanes Mariscal,M.A.

Publikováno v: Sanidad Militar v.72 n.1 2016
SciELO España. Revistas Científicas Españolas de Ciencias de la Salud
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Se reseñan los medicamentos evaluados y con dictamen positivo por comisión de expertos de la Agencia Española de Medicamentos y Productos Sanitarios o de la Agencia Europea del Medicamento hechos públicos en septiembre, octubre y noviembre de 201

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=RECOLECTA___::3a967e98b652bd7edfc5f9eee6f29510
http://scielo.isciii.es/scielo.php?script=sci_arttext&pid=S1887-85712016000100007

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