Rapid deterioration of a patient with mucopolysaccharidosis type I during interruption of enzyme replacement therapy-Reference-Cited by-同舟云学术

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Rapid deterioration of a patient with mucopolysaccharidosis type I during interruption of enzyme replacement therapy

Published:2007 Issue:16 Volume:143A Page:1925-1927
ISSN:1552-4825
Container-title:American Journal of Medical Genetics Part A
language:en
Short-container-title:Am. J. Med. Genet.

Author:

Węgrzyn Grzegorz,Tylki-Szymańska Anna,Liberek Anna,Piotrowska Ewa,Jakóbkiewicz-Banecka Joanna,Marucha Jolanta,Czartoryska Barbara,Węgrzyn Alicja

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Reference11 articles.

1. Effect of discontinuing of laronidase in a patient with mucopolysaccharidosis type I

2. Changes in serum chitotriosidase activity with cessation of replacement enzyme (cerebrosidase) administration in Gaucher disease

3. Cessation of enzyme replacement therapy in Gaucher disease

4. Enzyme-Replacement Therapy in Mucopolysaccharidosis I

5. Changes in hair morphology of mucopolysaccharidosis I patients treated with recombinant human α-L-iduronidase (laronidase, Aldurazyme)

Cited by 23 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Biomarkers for gene therapy clinical trials of lysosomal storage disorders;Molecular Therapy;2024-09

2. 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

3. 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

4. Invisible burden of COVID-19: enzyme replacement therapy disruptions;Journal of Pediatric Endocrinology and Metabolism;2021-04-05

5. Transcriptomic analyses suggest that mucopolysaccharidosis patients may be less susceptible to COVID‐19;FEBS Letters;2020-09-06

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