Maroteaux-Lamy Syndrome (Mucopolysaccharidosis Type VI) Medication

Updated: Mar 20, 2017
  • Author: Paul R Harmatz, MD; Chief Editor: Maria Descartes, MD  more...
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Medication

Medication Summary

Specific therapy for mucopolysaccharidosis type VI (MPS VI), also known as Maroteaux-Lamy syndrome, is just beginning to emerge, with recent clinical trials showing benefit from a recombinant DNA glycoprotein enzyme replacement therapy.

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Enzyme replacement therapy

Class Summary

Recombinant DNA variant of N -acetylgalactosamine 4-sulfatase has been shown to improve endurance of individuals with MPS VI. [12]

Galsulfase (Naglazyme)

Indicated for MPS VI, which is characterized by the absence or marked reduction of N -acetylgalactosamine 4-sulfatase; provides exogenous enzyme as treatment. Recombinant DNA glycoprotein (Chinese hamster ovary cell line) variant form of polymorphic human enzyme N -acetylgalactosamine 4-sulfatase. Clinical trials showed improvement in walking and stair-climbing capacity. Most patients in the clinical trials were pediatric patients; however, children < 5 y were not included.

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