Enzyme replacement therapy for mucopolysaccharidoses; past, present, and future-Reference-Cited by-同舟云学术

Enzyme replacement therapy for mucopolysaccharidoses; past, present, and future

Published:2019-08-27 Issue:11 Volume:64 Page:1153-1171
ISSN:1434-5161
Container-title:Journal of Human Genetics
language:en
Short-container-title:J Hum Genet

Author:

Chen Hui Hsuan,Sawamoto Kazuki,Mason Robert W.,Kobayashi Hironori,Yamaguchi Seiji,Suzuki Yasuyuki,Orii Kenji,Orii Tadao,Tomatsu Shunji

Publisher

Springer Science and Business Media LLC

Subject

Genetics(clinical),Genetics

Link

http://www.nature.com/articles/s10038-019-0662-9.pdf

Reference161 articles.

1. Coutinho MF, Prata MJ, Alves S. Mannose-6-phosphate pathway: a review on its role in lysosomal function and dysfunction. Mol Genet Metab. 2012;105:542–50.

2. Drugs@FDA: FDA approved drug products. accessdata.fda.gov. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=020057 . Accessed 26 Jul 2019.

3. Shawky RM, Elsayed SM. Treatment options for patients with Gaucher disease. Egypt J Med Hum Genet. 2016;17:281–5.

4. Semenza GL, Pyeritz RE. Respiratory complications of mucopolysaccharide storage disorders. Medicine. 1988;67:209–19.

5. Shih SL, Lee YJ, Lin SP, Sheu CY, Blickman JG. Airway changes in children with mucopolysaccharidoses: CT evaluation. Acta Radiol. 2002;43:40–3.

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