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Fig. 2 | Orphanet Journal of Rare Diseases

Fig. 2

From: Olipudase alfa enzyme replacement therapy for acid sphingomyelinase deficiency (ASMD): sustained improvements in clinical outcomes after 6.5 years of treatment in adults

Fig. 2

Assessment of Olipudase Alfa on Liver Volumes

A. Individual patient liver volumes by years of olipudase alfa treatment. Liver volumes were calculated by integrating cross-sectional magnetic resonance images and expressed as multiples of normal (MN) where normal liver volume was assumed to be 2.5% of body weight [18]. Severe and moderate hepatomegaly were defined as > 2.5 and > 1.25 to ≤ 2.5MN [18], respectively. Cutoffs of MN for severity of hepatomegaly are indicated by horizontal lines

B. Least square (LS) mean percent changes ± SE of the mean in liver volume from baseline over time. All p values are nominal

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