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Molecular Genetics and Metabolism
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February 1, 2024
Mucopolysaccharidosis type VII (Sly syndrome) - What do we know?
Christina L Grant, Jaime López-Valdez, Deborah Marsden, et al.
Journal of Pediatric Psychology
|
September 7, 2004
Brief report: Predictors of parenting stress among parents of children with biochemical genetic disorders
Susan E Waisbren, Michelle Rones, Catherine Y Read, et al.
Molecular Genetics and Metabolism
|
June 28, 2015
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, et al.
Molecular Genetics and Metabolism
|
June 22, 2020
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, et al.
Clinical Biochemistry
|
September 14, 2012
Recurrent unexplained hyperammonemia in an adolescent with arginase deficiency
Yan Zhang, Yuval E Landau, David T Miller, et al.
Current Medical Research and Opinion
|
September 18, 2024
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, et al.
Molecular Genetics and Metabolism Reports
|
July 7, 2023
Growth patterns in patients with mucopolysaccharidosis VII
Adriana M Montaño, Agnieszka Różdżyńska-Świątkowska, Agnieszka Jurecka, et al.
Molecular Genetics and Metabolism
|
April 23, 2022
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, et al.
Pediatrics
|
May 3, 2008
Spectrum of medium-chain acyl-CoA dehydrogenase deficiency detected by newborn screening
Ho-Wen Hsu, Thomas H Zytkovicz, Anne Marie Comeau, et al.
Molecular Genetics and Metabolism
|
March 25, 2022
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, et al.
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of 4
Search research articles
Search
Showing results (11-20 of 32) with videos related to
Sort By:
Page
of 4
Molecular Genetics and Metabolism
|
February 1, 2024
Mucopolysaccharidosis type VII (Sly syndrome) - What do we know?
Christina L Grant, Jaime López-Valdez, Deborah Marsden, et al.
Journal of Pediatric Psychology
|
September 7, 2004
Brief report: Predictors of parenting stress among parents of children with biochemical genetic disorders
Susan E Waisbren, Michelle Rones, Catherine Y Read, et al.
Molecular Genetics and Metabolism
|
June 28, 2015
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, et al.
Molecular Genetics and Metabolism
|
June 22, 2020
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, et al.
Clinical Biochemistry
|
September 14, 2012
Recurrent unexplained hyperammonemia in an adolescent with arginase deficiency
Yan Zhang, Yuval E Landau, David T Miller, et al.
Current Medical Research and Opinion
|
September 18, 2024
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, et al.
Molecular Genetics and Metabolism Reports
|
July 7, 2023
Growth patterns in patients with mucopolysaccharidosis VII
Adriana M Montaño, Agnieszka Różdżyńska-Świątkowska, Agnieszka Jurecka, et al.
Molecular Genetics and Metabolism
|
April 23, 2022
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, et al.
Pediatrics
|
May 3, 2008
Spectrum of medium-chain acyl-CoA dehydrogenase deficiency detected by newborn screening
Ho-Wen Hsu, Thomas H Zytkovicz, Anne Marie Comeau, et al.
Molecular Genetics and Metabolism
|
March 25, 2022
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, et al.
Page
of 4