Figure 3.

Tissue‐dependent effectiveness of the read‐through drug, ataluren (PTC124), in Cln1 ^R151X mice. Two‐month old Cln1 ^R151X male mice were treated with ataluren (10 mg/kg, i.p.) four times a day for 2 days. Control mice were treated with the vehicle of the drug. Immediately following the last injection on the second day, tissues were collected, and PPT1 enzyme activity was measured in the brain (cerebellum, cortex, striatum/thalamus, brainstem) and peripheral tissues (liver, heart, lung, kidney, skeletal muscle). Ataluren only increased PPT1 activity in the liver and muscle. Columns and bars represent mean ± S.E.M. (eight wild‐type and eight Cln1 ^R151X mice). Statistical significance was determined by unpaired t‐test.: *p=0.004 for liver, *p=0.009 for skeletal muscle.