Treatment-naive and post-treatment glucosylsphingosine (lyso-GL1) levels in a cohort of pediatric patients with Gaucher disease

Treatment-naive and post-treatment glucosylsphingosine (lyso-GL1) levels in a cohort of pediatric patients with Gaucher disease

Glucosylsphingosine (lyso-GL1) is a biomarker used to monitor disease and treatment response in Gaucher disease. Data from adults show that higher values of lyso-GL1 are associated with increased disease progression, however similar data in the pediatric population is lacking. In a cohort of pediatr...

Ausführliche Beschreibung

Gespeichert in:
Bibliographische Detailangaben
Veröffentlicht in:Molecular genetics and metabolism 2024-01, Vol.141 (1), p.107736-107736, Article 107736
Hauptverfasser: Rasmussen, Carly A., Quadri, Allegra, Vucko, Erika, Kim, Katherine, Hickey, Rachel, Baker, Joshua J., Charrow, Joel, Prada, Carlos E.
Format: Artikel
Sprache:eng
Schlagworte:
Biomarker
Gaucher disease
Glucosylsphingosine
Lysosomal storage diseases
Neuronopathic
Online-Zugang:Volltext
Tags: Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!
container_end_page 107736
container_issue 1
container_start_page 107736
container_title Molecular genetics and metabolism
container_volume 141
creator Rasmussen, Carly A.
Quadri, Allegra
Vucko, Erika
Kim, Katherine
Hickey, Rachel
Baker, Joshua J.
Charrow, Joel
Prada, Carlos E.
description Glucosylsphingosine (lyso-GL1) is a biomarker used to monitor disease and treatment response in Gaucher disease. Data from adults show that higher values of lyso-GL1 are associated with increased disease progression, however similar data in the pediatric population is lacking. In a cohort of pediatric patients, we present a relationship between lyso-GL1 value and Gaucher type, age, and treatment response. Data from this study may serve as a reference for providers monitoring children with Gaucher disease.
doi_str_mv 10.1016/j.ymgme.2023.107736
format Article
fullrecord <record><control><sourceid>proquest_cross</sourceid><recordid>TN_cdi_proquest_miscellaneous_2893839402</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><els_id>S1096719223003669</els_id><sourcerecordid>2893839402</sourcerecordid><originalsourceid>FETCH-LOGICAL-c359t-330877d5fd60d2a0ad211942224c24211b5d0fc0b7e55a7871f1aab9ad9f36b93</originalsourceid><addsrcrecordid>eNp90E1vEzEQBmALUdGS8guQkI_lsKk_9ssHDqiCgBSJS3u2vPZs4mh3vXi8QTnx13FJw5GTR553ZqSHkPecrTnj9f1hfRp3I6wFEzL_NI2sX5EbzlRdNILVry81V-KavEU8MMZ5pco35Fq2jDFZ1jfk92MEk0aYUjEZfwRqJkfngKlIlwbdDYsNeBpw3vtpF9BPQO-GE4Zis-Uf6QBHGJD6iRpqwz7ERENPZ3DepOgtnU3yeQ3SXz7t6cYsdg-ROo9gEG7JVW8GhHcv74o8ff3y-PCt2P7YfH_4vC2srFQqpGRt07iqdzVzwjDjBOeqFEKUVpS57irHesu6BqrKNG3De25Mp4xTvaw7JVfk7rx3juHnApj06NHCMJgJwoJatEq2UpXZckXkOWpjQIzQ6zn60cST5kw_y-uD_iuvn-X1WT5PfXg5sHQjuH8zF-oc-HQOZCw4eogabXax2SmCTdoF_98DfwDEbpcn</addsrcrecordid><sourcetype>Aggregation Database</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>2893839402</pqid></control><display><type>article</type><title>Treatment-naive and post-treatment glucosylsphingosine (lyso-GL1) levels in a cohort of pediatric patients with Gaucher disease</title><source>Elsevier ScienceDirect Journals</source><creator>Rasmussen, Carly A. ; Quadri, Allegra ; Vucko, Erika ; Kim, Katherine ; Hickey, Rachel ; Baker, Joshua J. ; Charrow, Joel ; Prada, Carlos E.</creator><creatorcontrib>Rasmussen, Carly A. ; Quadri, Allegra ; Vucko, Erika ; Kim, Katherine ; Hickey, Rachel ; Baker, Joshua J. ; Charrow, Joel ; Prada, Carlos E.</creatorcontrib><description>Glucosylsphingosine (lyso-GL1) is a biomarker used to monitor disease and treatment response in Gaucher disease. Data from adults show that higher values of lyso-GL1 are associated with increased disease progression, however similar data in the pediatric population is lacking. In a cohort of pediatric patients, we present a relationship between lyso-GL1 value and Gaucher type, age, and treatment response. Data from this study may serve as a reference for providers monitoring children with Gaucher disease.</description><identifier>ISSN: 1096-7192</identifier><identifier>EISSN: 1096-7206</identifier><identifier>DOI: 10.1016/j.ymgme.2023.107736</identifier><identifier>PMID: 38000346</identifier><language>eng</language><publisher>United States: Elsevier Inc</publisher><subject>Biomarker ; Gaucher disease ; Glucosylsphingosine ; Lysosomal storage diseases ; Neuronopathic</subject><ispartof>Molecular genetics and metabolism, 2024-01, Vol.141 (1), p.107736-107736, Article 107736</ispartof><rights>2023 Elsevier Inc.</rights><rights>Copyright © 2023 Elsevier Inc. All rights reserved.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c359t-330877d5fd60d2a0ad211942224c24211b5d0fc0b7e55a7871f1aab9ad9f36b93</citedby><cites>FETCH-LOGICAL-c359t-330877d5fd60d2a0ad211942224c24211b5d0fc0b7e55a7871f1aab9ad9f36b93</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://www.sciencedirect.com/science/article/pii/S1096719223003669$$EHTML$$P50$$Gelsevier$$H</linktohtml><link.rule.ids>314,776,780,3537,27901,27902,65306</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/38000346$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Rasmussen, Carly A.</creatorcontrib><creatorcontrib>Quadri, Allegra</creatorcontrib><creatorcontrib>Vucko, Erika</creatorcontrib><creatorcontrib>Kim, Katherine</creatorcontrib><creatorcontrib>Hickey, Rachel</creatorcontrib><creatorcontrib>Baker, Joshua J.</creatorcontrib><creatorcontrib>Charrow, Joel</creatorcontrib><creatorcontrib>Prada, Carlos E.</creatorcontrib><title>Treatment-naive and post-treatment glucosylsphingosine (lyso-GL1) levels in a cohort of pediatric patients with Gaucher disease</title><title>Molecular genetics and metabolism</title><addtitle>Mol Genet Metab</addtitle><description>Glucosylsphingosine (lyso-GL1) is a biomarker used to monitor disease and treatment response in Gaucher disease. Data from adults show that higher values of lyso-GL1 are associated with increased disease progression, however similar data in the pediatric population is lacking. In a cohort of pediatric patients, we present a relationship between lyso-GL1 value and Gaucher type, age, and treatment response. Data from this study may serve as a reference for providers monitoring children with Gaucher disease.</description><subject>Biomarker</subject><subject>Gaucher disease</subject><subject>Glucosylsphingosine</subject><subject>Lysosomal storage diseases</subject><subject>Neuronopathic</subject><issn>1096-7192</issn><issn>1096-7206</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2024</creationdate><recordtype>article</recordtype><recordid>eNp90E1vEzEQBmALUdGS8guQkI_lsKk_9ssHDqiCgBSJS3u2vPZs4mh3vXi8QTnx13FJw5GTR553ZqSHkPecrTnj9f1hfRp3I6wFEzL_NI2sX5EbzlRdNILVry81V-KavEU8MMZ5pco35Fq2jDFZ1jfk92MEk0aYUjEZfwRqJkfngKlIlwbdDYsNeBpw3vtpF9BPQO-GE4Zis-Uf6QBHGJD6iRpqwz7ERENPZ3DepOgtnU3yeQ3SXz7t6cYsdg-ROo9gEG7JVW8GhHcv74o8ff3y-PCt2P7YfH_4vC2srFQqpGRt07iqdzVzwjDjBOeqFEKUVpS57irHesu6BqrKNG3De25Mp4xTvaw7JVfk7rx3juHnApj06NHCMJgJwoJatEq2UpXZckXkOWpjQIzQ6zn60cST5kw_y-uD_iuvn-X1WT5PfXg5sHQjuH8zF-oc-HQOZCw4eogabXax2SmCTdoF_98DfwDEbpcn</recordid><startdate>202401</startdate><enddate>202401</enddate><creator>Rasmussen, Carly A.</creator><creator>Quadri, Allegra</creator><creator>Vucko, Erika</creator><creator>Kim, Katherine</creator><creator>Hickey, Rachel</creator><creator>Baker, Joshua J.</creator><creator>Charrow, Joel</creator><creator>Prada, Carlos E.</creator><general>Elsevier Inc</general><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope></search><sort><creationdate>202401</creationdate><title>Treatment-naive and post-treatment glucosylsphingosine (lyso-GL1) levels in a cohort of pediatric patients with Gaucher disease</title><author>Rasmussen, Carly A. ; Quadri, Allegra ; Vucko, Erika ; Kim, Katherine ; Hickey, Rachel ; Baker, Joshua J. ; Charrow, Joel ; Prada, Carlos E.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c359t-330877d5fd60d2a0ad211942224c24211b5d0fc0b7e55a7871f1aab9ad9f36b93</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2024</creationdate><topic>Biomarker</topic><topic>Gaucher disease</topic><topic>Glucosylsphingosine</topic><topic>Lysosomal storage diseases</topic><topic>Neuronopathic</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Rasmussen, Carly A.</creatorcontrib><creatorcontrib>Quadri, Allegra</creatorcontrib><creatorcontrib>Vucko, Erika</creatorcontrib><creatorcontrib>Kim, Katherine</creatorcontrib><creatorcontrib>Hickey, Rachel</creatorcontrib><creatorcontrib>Baker, Joshua J.</creatorcontrib><creatorcontrib>Charrow, Joel</creatorcontrib><creatorcontrib>Prada, Carlos E.</creatorcontrib><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><jtitle>Molecular genetics and metabolism</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Rasmussen, Carly A.</au><au>Quadri, Allegra</au><au>Vucko, Erika</au><au>Kim, Katherine</au><au>Hickey, Rachel</au><au>Baker, Joshua J.</au><au>Charrow, Joel</au><au>Prada, Carlos E.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Treatment-naive and post-treatment glucosylsphingosine (lyso-GL1) levels in a cohort of pediatric patients with Gaucher disease</atitle><jtitle>Molecular genetics and metabolism</jtitle><addtitle>Mol Genet Metab</addtitle><date>2024-01</date><risdate>2024</risdate><volume>141</volume><issue>1</issue><spage>107736</spage><epage>107736</epage><pages>107736-107736</pages><artnum>107736</artnum><issn>1096-7192</issn><eissn>1096-7206</eissn><abstract>Glucosylsphingosine (lyso-GL1) is a biomarker used to monitor disease and treatment response in Gaucher disease. Data from adults show that higher values of lyso-GL1 are associated with increased disease progression, however similar data in the pediatric population is lacking. In a cohort of pediatric patients, we present a relationship between lyso-GL1 value and Gaucher type, age, and treatment response. Data from this study may serve as a reference for providers monitoring children with Gaucher disease.</abstract><cop>United States</cop><pub>Elsevier Inc</pub><pmid>38000346</pmid><doi>10.1016/j.ymgme.2023.107736</doi><tpages>1</tpages></addata></record>
fulltext fulltext
identifier ISSN: 1096-7192
ispartof Molecular genetics and metabolism, 2024-01, Vol.141 (1), p.107736-107736, Article 107736
issn 1096-7192
1096-7206
language eng
recordid cdi_proquest_miscellaneous_2893839402
source Elsevier ScienceDirect Journals
subjects Biomarker
Gaucher disease
Glucosylsphingosine
Lysosomal storage diseases
Neuronopathic
title Treatment-naive and post-treatment glucosylsphingosine (lyso-GL1) levels in a cohort of pediatric patients with Gaucher disease
url https://sfx.bib-bvb.de/sfx_tum?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2025-01-30T10%3A24%3A28IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-proquest_cross&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.genre=article&rft.atitle=Treatment-naive%20and%20post-treatment%20glucosylsphingosine%20(lyso-GL1)%20levels%20in%20a%20cohort%20of%20pediatric%20patients%20with%20Gaucher%20disease&rft.jtitle=Molecular%20genetics%20and%20metabolism&rft.au=Rasmussen,%20Carly%20A.&rft.date=2024-01&rft.volume=141&rft.issue=1&rft.spage=107736&rft.epage=107736&rft.pages=107736-107736&rft.artnum=107736&rft.issn=1096-7192&rft.eissn=1096-7206&rft_id=info:doi/10.1016/j.ymgme.2023.107736&rft_dat=%3Cproquest_cross%3E2893839402%3C/proquest_cross%3E%3Curl%3E%3C/url%3E&disable_directlink=true&sfx.directlink=off&sfx.report_link=0&rft_id=info:oai/&rft_pqid=2893839402&rft_id=info:pmid/38000346&rft_els_id=S1096719223003669&rfr_iscdi=true