Tralesinidase alfa stabilizes brain atrophy in patients with MPS IIIB.
atrophy, MPS IIIB
ATROPHY
CGMV is a measure of the region of the brain most affected by atrophy in patients with MPS IIIB within the age range included in Study 250-201 (Participants enrolled in Study 250-201 lost, on average, 58 mL (median 68 mL) of CGMV from baseline to week 24, but lost only 2 mL from week 24 to week 48, for a total loss of 60 mL (median, 63 mL) from week 1 to week 48 (Nineteen of 22 patients treated with tralesinidase alfa had a total cerebellar volume within normal range at baseline (