Fig. 2: Ataluren treatment improved myeloid maturation in bone marrow.

Immunophenotype of myeloid lineage was analyzed in whole bone marrow aspirates by flow cytometry (n = 3, mean age 16.3, range 13–20). Analysis of bone marrow aspirates obtained from UPN26 (a), UPN58 (b), UPN74 (c) before (0) and after 6 months of ataluren treatment (6 m). Granulocytes (PMN, white) were detected by morphological analyses using CD45 as leukocyte marker. PMN were gated, and promyelocytes (light blue), myelocytes (green), metamyelocytes (orange), and neutrophils (yellow) were detected, staining CD16 and CD11b.