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Retrospective chart review of urinary glycosaminoglycan excretion and long-term clinical outcomes of enzyme replacement therapy in patients with mucopolysaccharidoses.
Jones, Simon A; Marsden, Deborah; Koutsoukos, Tony; Sniadecki, Jennifer; Tylee, Karen; Phillippo, Sarah; Kakkis, Emil.
Affiliation
  • Jones SA; Manchester University Hospitals NHS Foundation Trust, St Mary's Hospital, Manchester, UK. Electronic address: simon.jones@mft.nhs.uk.
  • Marsden D; Ultragenyx Pharmaceutical Inc., Novato, CA, USA.
  • Koutsoukos T; Ultragenyx Pharmaceutical Inc., Novato, CA, USA.
  • Sniadecki J; Ultragenyx Pharmaceutical Inc., Novato, CA, USA.
  • Tylee K; Manchester University Hospitals NHS Foundation Trust, St Mary's Hospital, Manchester, UK.
  • Phillippo S; Manchester University Hospitals NHS Foundation Trust, St Mary's Hospital, Manchester, UK.
  • Kakkis E; Ultragenyx Pharmaceutical Inc., Novato, CA, USA.
Mol Genet Metab ; 130(4): 255-261, 2020 08.
Article in En | MEDLINE | ID: mdl-32563631
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Biomarkers / Mucopolysaccharidosis II / Mucopolysaccharidosis I / Mucopolysaccharidosis VI / N-Acetylgalactosamine-4-Sulfatase / Enzyme Replacement Therapy / Glycosaminoglycans Type of study: Observational_studies / Prognostic_studies / Risk_factors_studies Limits: Child / Child, preschool / Female / Humans / Infant / Male / Newborn Language: En Journal: Mol Genet Metab Journal subject: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Year: 2020 Document type: Article Country of publication: Estados Unidos
Fulltext
Add to My VHL
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PubMed Links
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Biomarkers / Mucopolysaccharidosis II / Mucopolysaccharidosis I / Mucopolysaccharidosis VI / N-Acetylgalactosamine-4-Sulfatase / Enzyme Replacement Therapy / Glycosaminoglycans Type of study: Observational_studies / Prognostic_studies / Risk_factors_studies Limits: Child / Child, preschool / Female / Humans / Infant / Male / Newborn Language: En Journal: Mol Genet Metab Journal subject: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Year: 2020 Document type: Article Country of publication: Estados Unidos