Effect of rapid cessation of enzyme replacement therapy: A report of 5 cases and a review of the literature-Reference-Cited by-同舟云学术

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Effect of rapid cessation of enzyme replacement therapy: A report of 5 cases and a review of the literature

Published:2012-11 Issue:3 Volume:107 Page:508-512
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Jurecka Agnieszka,Żuber Zbigniew,Opoka-Winiarska Violetta,Węgrzyn Grzegorz,Tylki-Szymańska Anna

Funder

Polish MPS Society (‘Stowarzyszenie Chorych na Mukopolisacharydoze i Choroby Rzadkie’)

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference18 articles.

1. Treatment of lysosomal storage disorders: progress with enzyme replacement therapy;Rohrbach;Drugs,2007

2. Effect of discontinuing of laronidase in a patient with mucopolysaccharidosis type I;Anbu;J. Inherit. Metab. Dis.,2006

3. Rapid deterioration of a patient with mucopolysaccharidosis type I during interruption of enzyme replacement therapy;Węgrzyn;Am. J. Med. Genet. A,2007

4. Expert opinion on temporary treatment recommendations for Fabry disease during the shortage of enzyme replacement therapy (ERT) Mol;Linthorst;Mol. Genet. Metab.,2011

5. Klinische Tests an Knochen Gelenken und Muskeln;Buckup,1995

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2. Management of MPS-I using ERT: The Egyptian experience;2023-11-16

3. Safety outcomes and patients’ preferences for home-based intravenous enzyme replacement therapy (ERT) in pompe disease and mucopolysaccharidosis type I (MPS I) disorder: COVID-19 and beyond;Orphanet Journal of Rare Diseases;2023-10-27

4. Long-term experience with idursulfase beta (Hunterase) in two adolescent patients with MPS II: A case series;Molecular Genetics and Metabolism Reports;2023-09

5. Expert-opinion-based guidance for the care of children with lysosomal storage diseases during the COVID-19 pandemic: An experience-based Turkey perspective;Frontiers in Public Health;2023-01-30

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