Summary

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• Vimizim (elosulfase alfa) is indicated for the treatment of patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).
• This summary is based on the review of one systematic review(s)/meta-analysis(es). ^[1]
• Urinary Keratan Sulfate (uKS): The ERT group showed a reduction in uKS levels compared to the placebo group, with a mean difference of -0.260, indicating improvement in the biochemical marker for MPS IVA.
• Physical Performance: The ERT group demonstrated slight improvements in the 6-Minute Walk Test (mean difference: -0.102), 3-Minute Stair Climb Test (mean difference: -0.182), and lung function measures including Forced Vital Capacity (mean difference: -0.587) and FEV1 (mean difference: -0.293).
• Quality of Life and Mobility: Patients on ERT experienced improvements in self-care abilities (mean difference: -0.360), reduced need for caregiver assistance (mean difference: -0.408), and enhanced maximal voluntary ventilation (mean difference: -0.311).
• There is no safety information available in the reviewed studies.
• There is no population types or subgroups information available in the reviewed studies.