case_images: PMC11743810_01
case_images: Changes from baseline in (A) FVC % predicted, (B) 6MWT distance, and (C) QMFT total score in Patient 1 ( ) and the mean (SE) changes in the avalglucosidase alfa-arm ( ) from the COMET trial.
case_images: gr1.jpg
case_images: PMC11743810_01_gr1.jpg
case_images: f0005
case_images: In contrast, the overall population showed a gradual improvement over this timeframe (Fig. 1A). Changes in 6MWT also showed a decline followed by improvement at week 49, which eclipsed the gradual improvement seen in the overall population (Fig. 1B). The change from baseline to week 49 in QMFT total score in this Japanese patient (4.0) was similar to mean values of the overall population, which showed an improvement of approximately 5 points (Fig. 1C). This patient developed treatment-emergent ADA, with an intermediate peak titer (3200, Supplementary Fig. 1), and also developed neutralizing antibodies between week 13 and 25 that inhibited avalglucosidase alfa catalytic activity. These neutralizing antibodies became negative between week 29 and 49.
case_images: Change in (A) CK and (B) urinary Hex4 in Patient 1 ( ) and the mean (SD) changes in the avalglucosidase alfa-arm ( ) from the COMET trial.
case_images: gr2.jpg
case_images: PMC11743810_03_gr2.jpg
case_images: f0010
case_images: PMC11743810_02
case_images: Change in (A) CK and (B) urinary Hex4 in Patient 1 ( ) and the mean (SD) changes in the avalglucosidase alfa-arm ( ) from the COMET trial.
case_images: gr2.jpg
case_images: PMC11743810_03_gr2.jpg
case_images: f0010
case_images: Change in CK and urinary Hex4 in (A) Patient 2 and (B) Patient 3 ( ) together with mean (SD) changes in the overall population in each cohort ( ) from the Mini-COMET trial.
case_images: gr3.jpg
case_images: PMC11743810_03_gr3.jpg
case_images: f0015
case_images: Changes in CK and urinary Hex4 were also similar to mean values for the overall population (Fig. 3B).
case_images: PMC11743810_03
case_images: Change in (A) CK and (B) urinary Hex4 in Patient 1 ( ) and the mean (SD) changes in the avalglucosidase alfa-arm ( ) from the COMET trial.
case_images: gr2.jpg
case_images: PMC11743810_03_gr2.jpg
case_images: f0010
case_images: Change in CK and urinary Hex4 in (A) Patient 2 and (B) Patient 3 ( ) together with mean (SD) changes in the overall population in each cohort ( ) from the Mini-COMET trial.
case_images: gr3.jpg
case_images: PMC11743810_03_gr3.jpg
case_images: f0015
case_images: Changes in CK and urinary Hex4 were also similar to mean values for the overall population (Fig. 3B).