Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study.

J Inherit Metab Dis · 2014 · PMC4206772 · PMID 24810369

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failed to achieve significanceno p-value reported

The group composite efficacy measure, comprising 6MWT, 3MSCT, and MVV measurements, reflected the positive trends from the 6MWT and MVV endpoints but, due to small effects on 3MSCT, failed to achieve significance.

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