Table 2 Spearman correlation coefficients between changes in plasma lyso-Gb₃ and changes in LVMi, eGFR, pain, and KIC Gb₃ at specific timepoints during migalastat treatment.

		Overall (N = 97)		ERT-naive (n = 48)		ERT-experienced (n = 49)		Males (n = 37)		Females (n = 60)		Age ≤ 40 years (n = 31)		Age > 40 years (n = 66)
Parameter	Visit	n^a	P value^b	n^a	P value^b	n^a	P value^b	n^a	P value^b	n^a	P value^b	n^a	P value^b	n^a	P value^b
LVMi	Month 12	55	0.9283	17	0.5164	38	0.5311	21	0.4506	34	0.2742	16	0.4782	39	0.9254
	Month 18	44	0.5210	10	0.6515	34	0.9133	19	0.0663	25	0.4538	8	0.2604	36	0.1185
	Month 24	13	0.8164	13	0.8164	0	NA	3	0.6667	10	0.1921	6	0.1108	7	0.2939
	Month 30	27	0.2404	0	NA	27	0.2404	11	0.4669	16	0.1682	4	0.2000	23	0.3144
	Month 36	3	0.6667	0	NA	3	0.6667	1	NA	2	NA	0	NA	3	0.6667
eGFR	Month 12	61	0.1294	18	0.9773	43	0.1338	23	0.6183	38	0.1072	18	0.8357	43	0.0189
	Month 18	52	0.1052	13	0.8166	39	0.3793	21	0.0320	31	0.6475	11	0.7092	41	0.0660
	Month 24	27	0.4925	16	0.8201	11	0.2981	8	0.2894	19	0.9943	10	0.2763	17	0.1743
	Month 30	37	0.5586	0	NA	37	0.5586	15	0.8298	22	0.4373	6	0.8717	31	0.4231
	Month 36	29	0.3506	0	NA	29	0.3506	11	0.7092	18	0.2795	4	0.6000	25	0.2165
Pain	Month 12	61	0.8523	18	0.6612	43	0.8123	23	0.2968	38	0.1402	18	0.9224	43	0.6802
	Month 18	48	0.1935	13	0.3920	35	0.4525	20	0.8846	28	0.1310	10	0.1004	38	0.5628
	Month 24	18	0.8782	16	0.7804	2	NA	6	0.3123	12	0.8353	8	0.9081	10	0.8633
	Month 30	29	0.3309	0	NA	29	0.3309	13	0.3157	16	0.5872	4	0.4000	25	0.4522
	Month 36	5	0.4925	0	NA	5	0.4925	2	NA	3	1.0000	1	NA	4	0.2000
KIC Gb₃^c	Month 6	18	0.0003	18	0.0003	NA	NA	5	0.0374	13	0.0707	10	0.0022	7	0.0208
	Month 12	17	0.0470	17	0.0470	NA	NA	4	0.6000	13	0.7208	10	0.1076	8	0.4821

eGFR estimated glomerular filtration rate, ERT enzyme replacement therapy, Gb₃ globotriaosylceramide, KIC kidney interstitial capillary, LVMi left ventricular mass index, lyso-Gb₃ globotriaosylsphingosine, NA not applicable.
^an indicates number of patients with values for both lyso-Gb₃ and the other assessment at the specified timepoint.
^bP values represent Spearman correlations.
^cKIC Gb₃ data were derived from FACETS only.

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