TABLE 2.

Disease Responses in GLA and GSD Patients on Sirolimus Treatment

Response Category	GLA, n (%)	GSD, n (%)	GLA and GSD, n (%)
Overall
Improvement	12 (92)	3 (60)	15 (83)
No improvement	1 (8)	2 (40)	3 (17)
Stable disease	1 (8)	1 (20)	2 (11)
Progressive disease	0	1 (20)	1 (6)
Clinical status
Improvement	12 (92)	3 (60)	15 (83)
No improvement	1 (8)	2 (40)	3 (17)
Stable disease	1 (8)	2 (40)	3 (17)
Progressive disease	0	0	0
Quality of life
Improvement	11 (85)	3 (60)	14 (78)
No improvement	2 (15)	2 (40)	4 (22)
Stable disease	2 (15)	1 (20)	3 (17)
Progressive disease	0	1 (20)	1 (6)
Radiologic imaging
Improvement	4 (31)	1 (20)	5 (28)
No improvement	9 (69)	4 (80)	13 (72)
Stable disease	9 (69)	4 (80)	13 (72)
Progressive disease	0	0	0