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Elosulfase alfa (VIMIZIM®)

Morquio syndrome (MPS IV)

2 mg/kg weekly over 3-4 hours.(194-196)

Must be diluted with sodium chloride 9 mg/ml (0.9%) solution for injection to a final volume of 100 ml or 250 ml (based on the patient’s weight, ≥25 kg diluted in 250 ml while <25 kg diluted in 100 ml) prior to infusion and delivered intravenously.
5 ml, single-use vials.

IV

Hypersensitivity, reactions, fever, chills, fatigue, nausea, vomiting and headache.

1b

194- Burton BK, Berger KI, Lewis GD, Tarnopolsky M, Treadwell M, Mitchell JJ, Muschol N, Jones SA, Sutton VR, Pastores GM, Lau H, Sparkes, R, Genter F, Shaywitz AJ, Harmatz P. Safety and physiological effects of two different doses of elosulfase alfa in patients with Morquio a syndrome: a randomized, double-blind, pilot study. Am J Med Genet A. 2015;167A:2272–81.

195. Hendriksz CJ, Parini R, AlSayed M, Raiman J, Giugliani R, Mitchell J, Burton BK, Guelbert N, Stewart FJ, Hughes DA, Matousek R, Hawley SM, Decker C, Harmatz PR. Impact of long-term elosulfase alfa on activities of daily living in patients with Morquio A syndrome in an open-label, multi-center, phase 3 extension study. Mol Genet Metab. 2018;123:8.

196. Hendriksz C, Santra S, Jones SA, Geberhiwot T, Jesaitis L, Long B, Qi Y, Hawley SM, Decker C. Safety, immunogenicity, and clinical outcomes in patients with Morquio A syndrome participating in 2 sequential open-label studies of elosulfase alfa enzyme replacement therapy (MOR002/MOR-100), representing 5 years of treatment. Mol Genet Metab.2018;123:479–87.

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