Table 2. Effect of AT2220 co-administration on rhGAA-mediated glycogen reduction (% change from Baseline) in GAA KO mice.

Tissues	−AT2220	+AT2220 (10 mg/kg)	+AT2220 (30 mg/kg)
Heart	−54±5*	−68±5*	−85±2*#
Diaphragm	−48±6*	−49±5*	−60±6*#
Quadriceps	−20±4*	−45±3*#	−52±4*#
Gastrocnemius	−21±7	−41±4*	−45±5*#
Triceps	−10±4	−33±3*#	−24±3*#
Tongue	−44±3*	−54±6*#	−62±4*#

Twelve-week old male GAA KO mice were administered vehicle (water) or AT2220 via oral gavage once every other week for 8 weeks. Thirty minutes after each AT2220 oral administration, rhGAA (20 mg/kg) was administered via bolus tail vein injection. Mice were euthanized 21 days after the last (i.e., 4^th) rhGAA administration, and tissue glycogen levels were measured as described in ‘Materials and Methods’. Baseline glycogen levels in untreated GAA KO mice were 417±32, 340±24, 405±27, 353±21, 446±18, and 389±10 µg/mg protein in heart, diaphragm, quadriceps, gastrocnemius, triceps, and tongue, respectively, and in wild-type C57BL/6 mice were 23±2, 50±6, 40±5, 45±2, 35±7, and 32±2 µg/mg protein, respectively (mean±SEM of 7 mice). The data shown represent the percent glycogen change from baseline in each tissue as normalized between wild-type (0%) and GAA KO (100%) levels. Each value represents the mean±SEM of 12 mice. Statistically significant reductions were seen in glycogen levels compared to baseline (*p<0.05, t-test) and compared to rhGAA administration alone (#p<0.05, t-test).