Table 1.

Patient demographics: Response rate, age and SMA-related characteristics

	SMA subtype
	Total	I	II	III
Response rate
Total patient number	265	20	115	130
Enrolled respondents	189	12	73	104
Response rate [%]	71	60	63	80
Ratio of patients/parents among respondents [%]	52/48	0/100	30/70	74/26
Age of enrolled patients
Min. [years]	0	0	2	2
Max. [years]	73	7	56	73
Median [years]	19	1	11	33
Current motor function
N	177	9	69	99
Walk [%]	33	0	0	59
Sit [%]	47	11	65	38
None [%]	20	89	35	3
Wheelchair use
N	154	4	51	99
Always [%]	61	50	100	41
Sometimes [%]	14	0	0	21
None [%]	25	50	0	37
Feed tube use
N	177	9	69	99
Yes [%]	6	22	10	1
No [%]	94	78	90	99
Spine surgery
N	176	9	68	99
Yes [%]	23	0	22	8
No [%]	153	100	78	92
Non-invasive ventilation
N	174	9	69	96
Always [%]	1	0	1	0
Sometimes [%]	12	22	22	4
None [%]	87	78	77	96
Invasive ventilation
N	174	8	69	97
Always [%]	1	13	0	0
Sometimes [%]	0	0	0	0
None [%]	99	88	100	100

Differentiation by SMA subtypes (I = Werdnig-Hoffmann, II = intermediate, III = Kugelberg-Welander). This differentiation into subtypes is used for all following tables and figures. SMA-related characteristics of enrolled patients (motor function, spine surgery, use of wheelchair, feed tube use and ventilation) originate from the German SMA registry at the time of execution. Because of rounding, percentages might not add up to exactly 100 %. N = number of patients