Fig. 2.

ALT, AST, CK, and LDH after initiation of neoGAA therapy in the four patients.

Data on uGLC4 pre- and post-neoGAA were available for 3 of 4 patients. In Case #2, uGLC4 before neoGAA was 77 mmol/mol Creatinine (Cr) (range 49–122; nv 0.08–1.37 mmol/mol Cr); after 3 months of treatment uGLC4 excretion was 2.78 mmol/mol Cr. Case #3 had an uGlc4 of 9.51 mmol/mol Cr after 15 months of neoGAA, passing to 7.56 mmol/mol Cr after 18 months. In case #4, uGLC4 decreased by 10% after 9 months of therapy (from 28.9 mmol/mol Cr to 25.5 mmol/mol Cr.