Table 1.

Clinical features of the patients included in the study

P	G	Age at onset symptoms (years)	Age at ERT (years)	Age at Visit 0 (years)	Baseline walking aids	Baseline ventilation	Last visit walking aids	Last visit ventilation
1	F	38	47	50	N	N	Y (stick)	N
2	F	30	39	48	Y (stick, wheelchair)	Yes (non‐invasive)	Y (fully wheelchair)	Yes (non‐invasive)
3	F	—	—	26	N	N	N	N
4	F	49	59	63	Y (crouch)	N	Y (crouch)	N
5	F	30	42	45	N	N	N	N
6	F	27	47	51	N	N	N	N
7	M	62	67	66	N	Y (non‐invasive)	N	Y (non‐invasive)
8	F	48	52	59	Y (stick)	N	Y (stick)	Y (non‐invasive)
9	F	40	48	55	N	N	N	N
10	M	36	39	42	N	Y (non‐invasive)	N	Y (non‐invasive)
11	F	15	24	31	Y (Walker, wheelchair)	Y (non‐invasive)	Y (Walker, wheelchair)	Y (non‐invasive)
12	F	27	39	46	N	N	N	N
13	M	35	45	47	N	Y (non‐invasive)	N	Y (non‐invasive)
14	M	41	45	51	N	Y (non‐invasive)	N	Y (non‐invasive)
15	F	39	46	51	Y (crouch)	Y (non‐invasive)	Y (crouch)	Y (non‐invasive)
16	M	—	—	22	N	N	N	N
17	M	—	—	51	N	N	N	N
18	M	—	—	14	N	N	N	N
19	F	40	64	65	Y (stick)	N	Y (stick)	N
20	F	24	29	35	Y (crouch)	N	Y (crouch)	N
21	F	36	40	41	N	Y (non‐invasive)	N	Y (non‐invasive)
22	F	14	45	52	N	N	Y (stick)	N
23	M	22	57	64	Y (Stick)	Y (non‐invasive)	Y (stick)	Y (non‐invasive)
24	M	—	—	8	N	N	N	N
25	F	42	55	57	Y (Stick)	Y (non‐invasive)	Y (stick)	Y (non‐invasive)
26	M	35	43	46	N	Y (non‐invasive)	N	Y (non‐invasive)
27	M	34	51	51	N	N	N	Y (non‐invasive)
28	M	—	—	51	N	N	N	N
29	M	32	43	44	N	N	N	N
30	F	39	48	54	N	N	Y (stick)	N
31	M	—	—	12	N	N	N	N
32	M	—	—	51	N	N	N	N
33	M	38	43	42	Y (stick)	N	Y (stick)	N
34	F	20	22	21	N	N	N	N
35	F	—	—	35	N	N	N	N
36	F	24	40	29	N	N	N	N

F, female; G, gender; M, male; N, not needed; P, patient number; Y, yes.