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

Table 1 Demographic and other baseline characteristics (FAS)

	SA-ER	Placebo	Total
	(N = 10)	(N = 4)	(N = 14)
Sex (n [%])
Male	3 (30.0)	1 (25.0)	4 (28.6)
Female	7 (70.0)	3 (75.0)	10 (71.4)
Age (years)^a
Mean (SD)	41.3 (6.1)	37.0 (6.9)	40.1 (6.4)
Median	40.5	39.5	40.5
Min, Max	32, 50	27, 42	27, 50
Weight (kg)^b
Mean (SD)	58.22 (10.18)	55.93 (14.84)	57.56 (11.12)
Median	56.90	49.05	52.75
Min, Max	48.3, 78.3	47.5, 78.1	47.5, 78.3
Duration of illness (years)^c
Mean (SD)	10.8 (2.6)	10.5 (2.6)	10.7 (2.5)
Median	10.5	11.0	10.5
Min, Max	6, 15	7, 13	6, 15
Age of first GNE myopathy symptom (years)^d
Mean (SD)	31.4 (6.0)	27.5 (5.1)	30.3 (5.8)
Median	30.5	28.5	30.5
Min, Max	20, 41	21, 32	20, 41
UEC score at baseline (pattern: grip strength of 4 kg or less treated as 0 kg)
Mean (SD)	32.77 (12.37)	26.55 (8.74)	30.99 (11.50)
Median	27.53	27.03	27.53
Min, Max	19.60, 55.40	15.45, 36.70	15.45, 55.40
GNEM-FAS upper extremity score
Mean (SD)	27.4 (2.8)	28.0 (2.2)	27.6 (2.5)
Median	26.5	27.5	27.0
Min, Max	24, 32	26, 31	24, 32
Prior SA-ER administrated (n [%])
Yes	4 (40.0)	1 (25.0)	5 (35.7)

GNEM-FAS GNE myopathy-functional activity scale; Max maximum; Min minimum; N number of subjects in the treatment group; SA-ER sialic acid extended-release; SD standard deviation; UEC upper extremity composite
^aObtained informed consent
^bScreening
^cDuration of illness (years) = Year of informed consent—Year first symptom noted
^dAge of first GNE myopathy symptom (years) = Year first symptom noted—Year of birth

ISSN: 1750-1172