Sly Syndrome (Mucopolysaccharidosis Type VII) Medication

Updated: Nov 30, 2017
  • Author: Germaine L Defendi, MD, MS, FAAP; Chief Editor: Maria Descartes, MD  more...
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Medication

Medication Summary

The treatment of Sly syndrome (mucopolysaccharidosis type VII [MPS VII]) is symptomatic and supportive.

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Enzyme Replacement Therapy

Class Summary

Enzyme replacement therapy may provide clinically important benefits (eg, improved pulmonary function and walking ability, reduced excess carbohydrates stored in organs).

Vestronidase alfa-vjbk (Mepsevii)

Recombinant human lysosomal beta glucuronidase (GUS) is intended to provide exogenous GUS enzyme for uptake into cellular lysosome; mannose-6-phosphate (M6P) residues on the oligosaccharide chains allow binding of the enzyme to cell surface receptors, leading to cellular uptake of the enzyme, targeting to lysosomes and subsequent catabolism of accumulated GAGs in affected tissues.

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