Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study-Reference-Cited by-同舟云学术

Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study

Published:2014-05-09 Issue:6 Volume:37 Page:979-990
ISSN:0141-8955
Container-title:Journal of Inherited Metabolic Disease
language:en
Short-container-title:J Inherit Metab Dis

Author:

Hendriksz Christian J., ,Burton Barbara,Fleming Thomas R.,Harmatz Paul,Hughes Derralynn,Jones Simon A.,Lin Shuan-Pei,Mengel Eugen,Scarpa Maurizio,Valayannopoulos Vassili,Giugliani Roberto,Slasor Peter,Lounsbury Debra,Dummer Wolfgang

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Link

http://link.springer.com/content/pdf/10.1007/s10545-014-9715-6

Reference33 articles.

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4. Aslam R, van Bommel AC, Hendriksz CJ, Jester A (2013) Subjective and objective assessment of hand function in mucopolysaccharidosis IVa patients. JIMD Rep 9:59–65

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