Development of Multiexon Skipping Antisense Oligonucleotide Therapy for Duchenne Muscular Dystrophy

Table 1

Summary of clinical features of patients with an exon 45–55 deletion mutation of the DMD gene.

Patient ID	Age at onset of symptoms	Symptoms at onset	Clinical status	Dilated cardiomyopathy	Ambulant	Reference
1	2	Weakness	Mild BMD	not available	+	[58]
2	3.5	Weakness	Exercise intolerance	−	+	[58]
3	3.5	By chance	Asymptomatic	+	+	[58]
4	4	Weakness	Mild BMD	not available	+	[58]
5	4	Weakness	Mild BMD	not available	+	[58]
6	4	Fatigue, cramps after exercise	Mild BMD	−	+	[62]
7	4	Fatigue, mild difficulty in running	Mild BMD	−	+	[62]
8	6	Hyper-Ck-emia	Asymptomatic	−	+	[58]
9	8	Hyper-Ck-emia	Asymptomatic	−	+	[58]
10	9	Myoglobinuria	Mild BMD	−	+	[62]
11	12	Myalgia	Myalgia	not available	+	[58]
12	12	Exercise intolerance	Mild BMD	−	+	[58]
13	13	Muscle pain	Mild BMD	−	+	[58]
14	13	Hyper-Ck-emia	Mild BMD	−	+	[58]
15	19	Weakness	Mild BMD	−	+	[58]
16	26	Exertional dyspnea	No weakness and atrophy	+	+	[64]
17	36	Exertional dyspnea	No weakness and atrophy	+	+	[64]
18	40	Calf hypertrophy	Mild BMD	+	+	[58]
19	45	Weakness	Mild BMD	not available	+	[58]
20	49	Weakness	Mild BMD	not available	+	[58]
21	55	Walking difficulties	Mild BMD	−	+	[62]
22	59	Weakness	Mild BMD	−	+	[64]
23	69	Hyper-Ck-emia	Asymptomatic	−	+	[56]