Efficacy confirmation study of aceneuramic acid administration for GNE myopathy in Japan

Table 2 Linear mixed effect model of change from baseline in UEC score (FAS)

Treatment group	Analysis visit	n	LSM	Standard error	Difference in LSM ^a	95% CI for difference in LSM ^a
Treatment group	Analysis visit	n	LSM	Standard error	Difference in LSM ^a	Lower		Upper
SA-ER	Week 12	10	0.935	1.091	2.035	− 2.195	–	6.265
	Week 24	10	0.840	1.091	1.653	− 2.577	–	5.882
	Week 36	10	0.615	1.091	3.365	− 0.865	–	7.595
	Week 48	10	− 0.115	1.091	2.510	− 1.720	–	6.740
Placebo	Week 12	4	− 1.100	1.726	–	–	–	–
	Week 24	4	− 0.813	1.726	–	–	–	–
	Week 36	4	− 2.750	1.726	–	–	–	–
	Week 48	4	− 2.625	1.726	–	–	–	–

CI confidence interval; FAS full analysis set; LSM least square mean; n number of subjects analyzed; SA-ER sialic acid extended-release; UEC upper extremity composite
The linear mixed effect model includes treatment group, visit, treatment group*visit as fixed effects and subject as random effect. Degrees of freedom estimated using Kenward-Roger method. Not imputed, because there are not missing values at Week 48
^aSA-ER—Placebo

ISSN: 1750-1172