Correlations between tralesinidase alfa exposure and cognitive decline trajectory and plasma HS-NRE levels.
MPS IIIB
DISEASE
A treatment duration of longer than 48 weeks will be needed to establish whether there is a clinically meaningful benefit for cognition related to treatment with tralesinidase alfa. However, to elucidate whether there is early evidence of an effect of tralesinidase alfa on disease trajectory in patients with MPS IIIB, we explored correlations between a change in cognitive AEq and plasma drug exposure, defined as AUCA cumulative plasma HS-NRE concentration was calculated for each participant by integrating plasma HS-NRE concentrations measured every 4 weeks between weeks 12 and 36 of Study 250-201 Part 2. The average drug exposure, i.e., AUC