borderline significantp = 0.0480
Indeed, the significance of the difference in distributions of cognitive categories with idursulfase-IT versus no idursulfase-IT at 1 year was only borderline significant ( p = 0.0480).
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A post hoc analysis of Projected Retained Ability Scores (PRAS) for the longitudinal assessment of cognitive functioning in patients with neuronopathic mucopolysaccharidosis II receiving intrathecal idursulfase-IT.
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0.0480
0.0480
The sentences
DAS-II GCA scores within cognitive development categories The one patient with above-average cognitive growth rates in DAS-II GCA scores (who received idursulfase-IT in the randomized trial) had an increasing trend in GCA score over the 2 years; there was a relatively flatter trend in GCA scores among patients with average cognitive growth rates in DAS-II GCA scores, and a decreasing trend in GCA scores in patients with below-average cognitive growth rates in DAS-II GCA scores, plateauing cognitive functioning, or deteriorating cognitive functioning (both treatment groups; Fig. 3 A and B).
DAS-II GCA scores within cognitive development categories The one patient with above-average cognitive growth rates in DAS-II GCA scores (who received idursulfase-IT in the randomized trial) had an increasing trend in GCA score over the 2 years; there was a relatively flatter trend in GCA scores among patients with average cognitive growth rates in DAS-II GCA scores, and a decreasing trend in GCA scores in patients with below-average cognitive growth rates in DAS-II GCA scores, plateauing cognitive functioning, or deteriorating cognitive functioning (both treatment groups; Fig. 3 A and B).
Differences in the distribution of cognitive categories at 1 year did not reach statistical significance in the overall population but did reach statistical significance for the subpopulation aged younger than 6 years at phase 2/3 baseline in this post hoc analysis.