Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance-Reference-Cited by-同舟云学术

Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance

Published:2019-06-13 Issue:1 Volume:14 Page:
ISSN:1750-1172
Container-title:Orphanet Journal of Rare Diseases
language:en
Short-container-title:Orphanet J Rare Dis

Author:

Akyol Mehmet Umut, ,Alden Tord D.,Amartino Hernan,Ashworth Jane,Belani Kumar,Berger Kenneth I.,Borgo Andrea,Braunlin Elizabeth,Eto Yoshikatsu,Gold Jeffrey I.,Jester Andrea,Jones Simon A.,Karsli Cengiz,Mackenzie William,Marinho Diane Ruschel,McFadyen Andrew,McGill Jim,Mitchell John J.,Muenzer Joseph,Okuyama Torayuki,Orchard Paul J.,Stevens Bob,Thomas Sophie,Walker Robert,Wynn Robert,Giugliani Roberto,Harmatz Paul,Hendriksz Christian^ORCID,Scarpa Maurizio,

Funder

BioMarin Pharmaceutical

Publisher

Springer Science and Business Media LLC

Subject

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

Link

http://link.springer.com/content/pdf/10.1186/s13023-019-1074-9.pdf

Reference148 articles.

1. Muenzer J. Overview of the mucopolysaccharidoses. Rheumatology (Oxford). 2011;50(Suppl 5):v4–12.

2. James RA, Singh-Grewal D, Lee SJ, McGill J, Adib N, Australian Paediatric rheumatology G. Lysosomal storage disorders: a review of the musculoskeletal features. J Paediatr Child Health. 2016;52:262–71.

3. Leone A, Rigante D, Amato DZ, Casale R, Pedone L, Magarelli N, et al. Spinal involvement in mucopolysaccharidoses: a review. Childs Nerv Syst. 2015;31:203–12.