|AHFS/Drugs.com||Multum Consumer Information|
|Chemical and physical data|
|Molar mass||110.8 kg/mol|
Elosulfase alfa (trade name Vimizim) is a drug for the treatment of Morquio syndrome which is caused by a deficiency in the enzyme N-acetylgalactosamine-6-sulfatase. Elosulfase alfa is a synthetic version of this enzyme.
The drug is used in enzyme replacement therapy; a 2014 study confirmed it was effective on young patients with Morquio syndrome type A. Treatment with this medication was most effective upon respiratory symptoms, activities of daily living and growth, as confirmed in a 2015 paper.
- "FDA approves Vimizim to treat rare congenital enzyme disorder". Food and Drug Administration. Feb 14, 2014.
- Hendriksz, C. et. al (November 2014). "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". Journal of Inherited Metabolic Disease. 37 (6): 979–990. doi:10.1007/s10545-014-9715-6.
- Hendriksz, C. et. al (February 2015). "Multi-domain impact of elosulfase alfa in Morquio A syndrome in the pivotal phase III trial". Molecular Genetics and Metabolism. 114 (2): 178–185. doi:10.1016/j.ymgme.2014.08.012. PMID 25284089.
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