Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: a retrospective case series analysis of nine sibships-Reference-Cited by-同舟云学术

Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: a retrospective case series analysis of nine sibships

Published:2015-10-07 Issue:1 Volume:10 Page:
ISSN:1750-1172
Container-title:Orphanet Journal of Rare Diseases
language:en
Short-container-title:Orphanet J Rare Dis

Author:

Al-Sannaa Nouriya A.,Bay Luisa,Barbouth Deborah S.,Benhayoun Youssef,Goizet Cyril,Guelbert Norberto,Jones Simon A.,Kyosen Sandra Obikawa,Martins Ana Maria,Phornphutkul Chanika,Reig Celia,Pleat Rebecca,Fallet Shari,Ivanovska Holder Iva

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Genetics (clinical),General Medicine

Link

http://link.springer.com/content/pdf/10.1186/s13023-015-0344-4

Reference19 articles.

1. Beck M, Arn P, Giugliani R, Muenzer J, Okuyama T, Taylor J, et al. The natural history of MPS I: global perspectives from the MPS I Registry. Genet Med. 2014;16(10):759–65. doi: 10.1038/gim.2014.25 .

2. Muenzer J, Wraith JE, Clarke LA. Mucopolysaccharidosis I: management and treatment guidelines. Pediatrics. 2009;123(1):19–29. doi: 10.1542/peds.2008-0416 .

3. Clarke LA, Wraith JE, Beck M, Kolodny EH, Pastores GM, Muenzer J, et al. Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I. Pediatrics. 2009;123(1):229–40. doi: 10.1542/peds.2007-3847 .

4. Kakkis ED, Muenzer J, Tiller GE, Waber L, Belmont J, Passage M, et al. Enzyme-replacement therapy in mucopolysaccharidosis I. N Engl J Med. 2001;344(3):182–8. doi: 10.1056/NEJM200101183440304 .

5. Wraith JE, Beck M, Lane R, van der Ploeg A, Shapiro E, Xue Y, et al. Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human alpha-L-iduronidase (laronidase). Pediatrics. 2007;120(1):e37–46. doi: 10.1542/peds.2006-2156 .

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