Publications by authors named "Deborah Marsden"

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Clinical manifestations, healthcare resource utilization, and costs among patients with long-chain fatty acid oxidation disorders: a retrospective claims database analysis.
Erru Christy Yang, Zhenzhen Fang, Ruixin Tan, Yun Guo, Siyi He, Deborah Marsden

Curr Med Res Opin· November 2024

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Major clinical events and healthcare resource use among patients with long-chain fatty acid oxidation disorders in the United States: Results from LC-FAOD Odyssey program.
Erru Yang, Eliza Kruger, David Yin, Kieran Mace, Meghan Tierney, Deborah Marsden

Mol Genet Metab· May 2024

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Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring program.
Roberto Giugliani, Antonio Gonzalez-Meneses, Maurizio Scarpa, Barbara Burton, Raymond Wang, Deborah Marsden

Orphanet J Rare Dis· May 2024

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Mucopolysaccharidosis type VII (Sly syndrome) - What do we know?
Christina L Grant, Jaime López-Valdez, Deborah Marsden, Fatih Ezgü

Mol Genet Metab· March 2024

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Growth patterns in patients with mucopolysaccharidosis VII.
Adriana M Montaño, Agnieszka Różdżyńska-Świątkowska, Agnieszka Jurecka, Antonio Nino Ramirez, Lin Zhang, Deborah Marsden

Mol Genet Metab Rep· September 2023

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Estimating the Prevalence of Rare Diseases: Long-Chain Fatty Acid Oxidation Disorders as an Illustrative Example.
Eliza Kruger, Paul McNiven, Deborah Marsden

Adv Ther· July 2022

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Response to triheptanoin therapy in critically ill patients with LC-FAOD: Report of patients treated through an expanded access program.
Jerry Vockley, Gregory M Enns, Antonio Nino Ramirez, Camille L Bedrosian, Bridget Reineking, Deborah Marsden

Mol Genet Metab· June 2022

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Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in pediatric subjects < 5 years with mucopolysaccharidosis VII.
Heather A Lau, David Viskochil, Pranoot Tanpaiboon, Antonio Gonzalez-Meneses Lopez, Esmeralda Martins, Deborah Marsden

Mol Genet Metab· May 2022

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Impact of newborn screening on the reported incidence and clinical outcomes associated with medium- and long-chain fatty acid oxidation disorders.
Deborah Marsden, Camille L Bedrosian, Jerry Vockley

Genet Med· May 2021

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Retrospective chart review of urinary glycosaminoglycan excretion and long-term clinical outcomes of enzyme replacement therapy in patients with mucopolysaccharidoses.
Simon A Jones, Deborah Marsden, Tony Koutsoukos, Jennifer Sniadecki, Karen Tylee

Mol Genet Metab· August 2020

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Corrigendum to "The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII" [Mol Genet Metab 2020 Mar;129(3):219-227].
Raymond Y Wang, José Francisco da Silva Franco, Jaime López-Valdez, Esmeralda Martins, Vernon Reid Sutton, Deborah Marsden

Mol Genet Metab· August 2020

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The re-occurrence of cardiomyopathy in propionic acidemia after liver transplantation.
Gerard T Berry, Elizabeth D Blume, Ann Wessel, Tajinder Singh, Leah Hecht, Deborah Marsden

JIMD Rep· July 2020

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Urinary glycosaminoglycans as a potential biomarker for evaluating treatment efficacy in subjects with mucopolysaccharidoses.
Emil Kakkis, Deborah Marsden

Mol Genet Metab· May 2020

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The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII.
Raymond Y Wang, José Francisco da Silva Franco, Jaime López-Valdez, Esmeralda Martins, Vernon Reid Sutton, Deborah Marsden

Mol Genet Metab· March 2020

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Regulatory strategies for rare diseases under current global regulatory statutes: a discussion with stakeholders.
Andrew E Mulberg, Christina Bucci-Rechtweg, Joseph Giuliano, David Jacoby, Franklin K Johnson, Deborah Marsden

Orphanet J Rare Dis· February 2019

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Results from a 78-week, single-arm, open-label phase 2 study to evaluate UX007 in pediatric and adult patients with severe long-chain fatty acid oxidation disorders (LC-FAOD).
Jerry Vockley, Barbara Burton, Gerard T Berry, Nicola Longo, John Phillips, Deborah Marsden

J Inherit Metab Dis· January 2019

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Clinical, Biochemical, and Molecular Features in 37 Saudi Patients with Very Long Chain Acyl CoA Dehydrogenase Deficiency.
Abdulrahman Obaid, Marwan Nashabat, Majid Alfadhel, Ali Alasmari, Fuad Al Mutairi, Deborah Marsden

JIMD Rep· October 2017

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Newborn Screening for Lysosomal Storage Disorders: Quo Vadis?
Roy W A Peake, Deborah L Marsden, Olaf A Bodamer, Michael H Gelb, David S Millington

Clin Chem· November 2016

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Long-term major clinical outcomes in patients with long chain fatty acid oxidation disorders before and after transition to triheptanoin treatment--A retrospective chart review.
Jerry Vockley, Deborah Marsden, Elizabeth McCracken, Stephanie DeWard, Amanda Barone

Mol Genet Metab· July 2016

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Recurrent unexplained hyperammonemia in an adolescent with arginase deficiency.
Yan Zhang, Yuval E Landau, David T Miller, Deborah Marsden, Gerard T Berry

Clin Biochem· December 2012

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Newborn screening of lysosomal storage disorders.
Deborah Marsden, Harvey Levy

Clin Chem· July 2010

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Newborn screening.
Inderneel Sahai, Deborah Marsden

Crit Rev Clin Lab Sci· April 2009

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Commentary on a Delphi clinical practice protocol for the diagnosis and management of very long chain acyl-CoA dehydrogenase deficiency by Arnold et al.
Deborah Louise Marsden

Mol Genet Metab· March 2009

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Spectrum of medium-chain acyl-CoA dehydrogenase deficiency detected by newborn screening.
Ho-Wen Hsu, Thomas H Zytkovicz, Anne Marie Comeau, Arnold W Strauss, Deborah Marsden

Pediatrics· May 2008

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Development of a newborn screening follow-up algorithm for the diagnosis of isobutyryl-CoA dehydrogenase deficiency.
Devin Oglesbee, Miao He, Nilanjana Majumder, Jerry Vockley, Ayesha Ahmad, Deborah Marsden

Genet Med· February 2007

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