Review Article
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] |
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