First 1.5 years of pegvaliase clinic: Experiences and outcomes

We present Boston Children's Hospital's clinic model for pegvaliase therapy in adults with phenylketonuria (PKU) and clinical outcomes in 46 patients over the first 1.5 years of commercial therapy. Approximately 70% (18/26) of patients starting pegvaliase achieved blood phenylalanine (Phe)...

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Autores principales: Sacharow, Stephanie, Papaleo, Cassandra, Almeida, Kyla, Goodlett, Benjamin, Kritzer, Amy, Levy, Harvey, Martell, Leslie, Wessel, Ann, Viau, Krista
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2020
Materias:
Short Communication
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7256053/
https://www.ncbi.nlm.nih.gov/pubmed/32489881
http://dx.doi.org/10.1016/j.ymgmr.2020.100603
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author Sacharow, Stephanie
Papaleo, Cassandra
Almeida, Kyla
Goodlett, Benjamin
Kritzer, Amy
Levy, Harvey
Martell, Leslie
Wessel, Ann
Viau, Krista
author_facet Sacharow, Stephanie
Papaleo, Cassandra
Almeida, Kyla
Goodlett, Benjamin
Kritzer, Amy
Levy, Harvey
Martell, Leslie
Wessel, Ann
Viau, Krista
author_sort Sacharow, Stephanie
collection PubMed
description We present Boston Children's Hospital's clinic model for pegvaliase therapy in adults with phenylketonuria (PKU) and clinical outcomes in 46 patients over the first 1.5 years of commercial therapy. Approximately 70% (18/26) of patients starting pegvaliase achieved blood phenylalanine (Phe) <360 μmol/L, with an average of a 68 ± 24% decrease in blood Phe from baseline. All patients experienced at least minor side effects, but in most, management of the side effects allowed for treatment to continue.
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spelling pubmed-72560532020-06-01 First 1.5 years of pegvaliase clinic: Experiences and outcomes Sacharow, Stephanie Papaleo, Cassandra Almeida, Kyla Goodlett, Benjamin Kritzer, Amy Levy, Harvey Martell, Leslie Wessel, Ann Viau, Krista Mol Genet Metab Rep Short Communication We present Boston Children's Hospital's clinic model for pegvaliase therapy in adults with phenylketonuria (PKU) and clinical outcomes in 46 patients over the first 1.5 years of commercial therapy. Approximately 70% (18/26) of patients starting pegvaliase achieved blood phenylalanine (Phe) <360 μmol/L, with an average of a 68 ± 24% decrease in blood Phe from baseline. All patients experienced at least minor side effects, but in most, management of the side effects allowed for treatment to continue. Elsevier 2020-05-25 /pmc/articles/PMC7256053/ /pubmed/32489881 http://dx.doi.org/10.1016/j.ymgmr.2020.100603 Text en © 2020 The Authors http://creativecommons.org/licenses/by-nc-nd/4.0/ This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
spellingShingle Short Communication
Sacharow, Stephanie
Papaleo, Cassandra
Almeida, Kyla
Goodlett, Benjamin
Kritzer, Amy
Levy, Harvey
Martell, Leslie
Wessel, Ann
Viau, Krista
First 1.5 years of pegvaliase clinic: Experiences and outcomes
title First 1.5 years of pegvaliase clinic: Experiences and outcomes
title_full First 1.5 years of pegvaliase clinic: Experiences and outcomes
title_fullStr First 1.5 years of pegvaliase clinic: Experiences and outcomes
title_full_unstemmed First 1.5 years of pegvaliase clinic: Experiences and outcomes
title_short First 1.5 years of pegvaliase clinic: Experiences and outcomes
title_sort first 1.5 years of pegvaliase clinic: experiences and outcomes
topic Short Communication
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7256053/
https://www.ncbi.nlm.nih.gov/pubmed/32489881
http://dx.doi.org/10.1016/j.ymgmr.2020.100603
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