Higher dosing of alglucosidase alfa improves outcomes in children with Pompe disease: a clinical study and review of the literature-Reference-Cited by-同舟云学术

Higher dosing of alglucosidase alfa improves outcomes in children with Pompe disease: a clinical study and review of the literature

Published:2020-05 Issue:5 Volume:22 Page:898-907
ISSN:1098-3600
Container-title:Genetics in Medicine
language:en
Short-container-title:Genetics in Medicine

Author:

Khan Aleena A.,Case Laura E.,Herbert Mrudu,DeArmey Stephanie,Jones Harrison,Crisp Kelly,Zimmerman Kanecia,ElMallah Mai K.,Young Sarah P.,Kishnani Priya S.^ORCID

Publisher

Elsevier BV

Subject

Genetics (clinical)

Link

http://www.nature.com/articles/s41436-019-0738-0.pdf

Reference40 articles.

1. Hirschhorn R, Reuser A. Glycogen storage disease type II: acid alpha-glucosidase (acid maltase) deficiency. In: Scriver C, Beaudet A, Sly W, editors. The metabolic and molecular bases of inherited disease. New York: McGraw-Hill; 2001. p. 3389–3420.

Cited by 43 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. Optimizing clinical outcomes: The journey of twins with CRIM-negative infantile-onset Pompe disease on high-dose enzyme replacement therapy and immunomodulation;Molecular Genetics and Metabolism Reports;2024-12

2. Non-invasive optoacoustic imaging of glycogen-storage and muscle degeneration in late-onset Pompe disease;Nature Communications;2024-09-08

3. Avalglucosidase alfa in infantile-onset Pompe disease: A snapshot of real-world experience in Italy;Molecular Genetics and Metabolism Reports;2024-09

4. Clinical insight meets scientific innovation to develop a next generation ERT for Pompe disease;Molecular Genetics and Metabolism;2024-09

5. The Importance of Early Treatment of Inherited Neuromuscular Conditions;Journal of Neuromuscular Diseases;2024-03-05