Table 1 Number of patients with exon deletions amenable to exon-skipping therapy

44

Exons 17–43 (2); exons 19–43 (2); exons 35–43 (1); exons 38–43 (1); exons 40–43 (2); exon 41–43 (1); exon 43 (7); exon 45 (51); exons 45–54 (16); exons 45–56 (1)

81

45

Exons 12–44 (1); exons 18–44 (3); exon 44 (26); exon 46 (1); exons 46–47(28); exons 46–48 (13); exons 46–49 (5); exons 46–51 (16); exons 46–53 (3); exons 46–55 (3); exons 46–57 (2)

101

51

Exons 3–50 (1); exons 17–50 (1); exons 30–50 (1); exons 35–50 (1); exons 45–50 (44); exons 47–50 (5); exons 48–50 (38); exons 49–50 (37); exon 50 (18)

146

53

Exons 45–52 (48); exons 47–52 (6); exons 48–52 (36); exons 49–52 (30); exons 50–52 (8); exon 52 (22)^a

150