Summary

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• This summary is based on the review of one systematic review(s)/meta-analysis(es). ^[1]
• Urinary Keratan Sulfate (uKS) Reduction: Elosulfase alfa treatment resulted in a mean reduction of -0.260 in urinary keratan sulfate levels compared to the placebo group, indicating a decrease in this biomarker for Mucopolysaccharidosis type IVA (MPS IVA) (total participants: 550; 327 on enzyme replacement therapy (ERT) treatment vs. 223 on placebo).
• Improvement in Physical Function: The 6-Minute Walk Test and 3-Minute Stair Climb Test showed mean differences of -0.102 and -0.182, respectively, suggesting enhanced walking ability and stair climbing capacity in the elosulfase alfa treatment group.
• Enhanced Self-Care and Respiratory Function: The Self-Care MPS-Health Assessment Questionnaire showed a mean difference of -0.360, reflecting better self-care abilities, while improvements in respiratory function were indicated by differences in Forced Vital Capacity (-0.587), First Second of Forced Expiration (-0.293), and Maximal Voluntary Ventilation (-0.311) in the elosulfase alfa group compared to placebo.
• There is no safety information available in the reviewed studies.
• There is no population types or subgroups information available in the reviewed studies.