Table 1.

Paired-sample t-tests assessing mean differences in measured biomarker levels over 42 days in seven Fabry patients at two different collection time points (morning and evening). Means ± standard deviation are shown. For statistically significant results, p-values are highlighted in gray or in black if the highest mean concentration value was measured in the morning or the evening, respectively.

	Participant 1			Participant 2			Participant 3			Participant 4			Participant 5			Participant 6			Participant 7
	M	E	p	M	E	p	M	E	p	M	E	p	M	E	p	M	E	p	M	E	p
Gb3	373 ± 128	468 ± 261	<0.050	28 ± 8	44 ± 23	<0.001 †	12 ± 5	10 ± 3	<0.050	33 ± 9	38 ± 11	<0.050	20 ± 8	29 ± 12	<0.001 †	49 ± 17	66 ± 52	0.059	4 ± 2	6 ± 4	0.060
Lyso-Gb3	188 ± 86	295 ± 94	<0.001 †	59 ± 27	111 ± 61	<0.001 †	22 ± 9	21 ± 7	0.525	62 ± 12	81 ± 27	<0.050	27 ± 11	34 ± 13	<0.050	82 ± 14	119 ± 36	<0.001 †	12 ± 5	17 ± 6	<0.001 †
Lyso-Gb3 (−28)	204 ± 46	228 ± 33	<0.050	23 ± 7	30 ± 7	<0.001 †	14 ± 5	12 ± 4	<0.050	64 ± 8	55 ± 16	0.001 †	13 ± 3	18 ± 6	<0.001 †	42 ± 9	40 ± 12	0.224	5 ± 2	6 ± 3	0.208
Lyso-Gb3 (−12)	1620 ± 193	1576 ± 281	0.282	77 ± 17	77 ± 22	0.894	65 ± 15	53 ± 13	<0.001 †	224 ± 33	226 ± 43	0.831	56 ± 14	50 ± 15	<0.050	99 ± 15	93 ± 30	0.224	50 ± 7	46 ± 9	<0.050
Lyso-Gb3 (−2)	585 ± 126	689 ± 95	<0.001 †	35 ± 10	44 ± 11	<0.001 †	32 ± 10	27 ± 9	<0.050	122 ± 15	118 ± 30	0.393	24 ± 5	33 ± 10	<0.001 †	77 ± 10	92 ± 26	0.001 †	9 ± 3	11 ± 5	0.135
Lyso-Gb3 (+14)	894 ± 138	855 ± 188	<0.050	24 ± 6	23 ± 6	0.105	26 ± 6	23 ± 6	<0.050	94 ± 14	93 ± 22	0.457	14 ± 3	14 ± 3	0.768	27 ± 5	24 ± 12	0.280	8 ± 2	9 ± 4	0.457
Lyso-Gb3 (+16)	5524 ± 1017	5246 ± 1351	<0.050	204 ± 43	206 ± 45	0.760	215 ± 39	182 ± 42	<0.001 †	797 ± 109	800 ± 196	0.927	115 ± 27	110 ± 21	0.279	267 ± 40	283 ± 73	0.234	67 ± 11	72 ± 20	0.094
Lyso-Gb3 (+34)	2892 ± 440	2622 ± 532	0.001	121 ± 26	118 ± 28	0.527	125 ± 25	104 ± 26	<0.001 †	315 ± 60	316 ± 98	0.797	80 ± 18	72 ± 13	<0.050	146 ± 25	142 ± 39	0.609	60 ± 11	65 ± 20	0.137
Lyso-Gb3 (+50)	4973 ± 711	4613 ± 913	<0.050	69 ± 15	68 ± 20	0.779	99 ± 20	77 ± 18	<0.001 †	144 ± 27	144 ± 37	0.847	39 ± 9	34 ± 8	<0.050	70 ± 13	59 ± 20	<0.050	51 ± 7	50 ± 11	0.583

^† indicates that statistical significance was still valid after correction for multiple comparisons using the Holm–Šídák procedure (63 comparisons); M: morning collection; E: evening collection. All participants were under ERT except for participant no. 1 who was an ERT-naïve patient. Patients 1–4 were males, whereas patients 5–7 were females. For lyso-Gb₃ analogues, the mass difference in Da compared to lyso-Gb₃ is indicated in brackets.