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Electron microscopy examination of SM accumulation and clearance from hepatocytes. A, The presence of large SM masses mixed with lipofuscin (L) within hepatocytes at baseline in patient 5. Smaller whorls of SM are also present (black arrows). B, The reduction of SM after 26 weeks of olipudase alfa treatment. Black arrows indicate residual SM. Electron microscopy scale bars=1 μm.
