Children and adolescent patients
BBS
For children and adolescents (< 18 years old; n = 9) with BBS, mean (range) PedsQL total score at active treatment baseline was 67.2 (33.7–90.2; Table Overall, 4 of 9 (44.4%) children experienced impairment in HRQOL at active treatment baseline (mean [SD] total score, 47.8 [10.9], n = 4). All children with HRQOL impairment based on PedsQL at active treatment baseline (n = 4) experienced clinically meaningful improvement after 52 weeks of treatment (Fig. Change in PedsQL and IWQOL-Lite scores after 52 weeks. Spaghetti plots of individual patient HRQOL course in 