Table 2. Liver fibrosis scores and liver function assessments.

Four of five patients showed reductions in ALT and AST levels after 26 weeks of treatment. The fifth patient, patient 3, showed no change in ALT; an increase in AST from 20 to 26 IU/L remained within the normal reference range. Interestingly, the patients with the highest histopathologic sphingomyelin burden at baseline (patient 1, 2 and 4), also had the highest ALT and AST levels, whereas the patients with the lowest histopathologic sphingomyelin levels (patients 3 and 5) also had the lowest baseline ALT and AST. (‘H’ indicates values outside of normal reference ranges: ALT 1-53 IU/L, AST 1-50 IU/L)

Patient number	Fibrosis score		ALT (IU/L)		AST (IU/L)
	Baseline	26 weeks	Baseline	26 weeks	Baseline	26 weeks
1	3*	4*	96 (H)	22	94 (H)	28
2	1	1	41	15	40	23
3	0	1	16	16	20	26
4	2*	3	38	13	35	21
5	2	2	18	15	18	16

^*The length of the gross specimen received in 10% neutral buffered formalin was less than the 1.5 cm minimum length specified in the Study Operations Manual which is recommended to minimize sampling error (34).