From: Long term treatment with ataluren—the Swedish experience
Patient ID | Age at onset | Corticosteroid treatment (Yes/No)/Type | Age at start of corticosteroid treatment (years) | Age at ataluren start (years) | Age at last follow-up (years) | Ambulation status at last follow-up | Age at loss of ambulation (years) | Cumulative use of ataluren (years)a | Age at discontinuation of ataluren (years) | Genotype |
---|---|---|---|---|---|---|---|---|---|---|
p1 | 4 | Y/P | 4 | 6.2 | 17.4 | Non-ambulatory | 12.9 | 8.3 | NA | exon 19, p.Lys773* |
p2 | n.a | Y/P | 4 | 14.4 | 26.5 | Non-ambulatory | 17.1 | 8.6 | 25.5 | Exon 30, p.Gln1370* |
p3 | 1.5 | Y/P | 4 | 5.2 | 17.3 | Non-ambulatory | 12.4 | 6.9 | 14.7 | exon 52, p.Arg2553* |
p4 | 1.5 | Y/D | 4 | 11.5 | 23.3 | Non-ambulatory | 13.5 | 6.3 | 20.5 | Exon 39, p.L1834X |
p5 | 1.5 | Y/P | 4.5 | 8.4 | 20.3 | Non-ambulatory | 18.1 | 9.4 | NA | exon 35, p.R1666* |
p6 | n.a | Y/P | Preschoolb | 10.3 | 16.2 | Ambulatory | NA | 5.9 | NA | exon 20, p.Ser861* |
p7 | 4 | Y/D | 5 | 10.2 | 16.0 | Non-ambulatory | 12.9 | 4.0 | 14.2 | exon 58, p.Glu2886* |
p8 | 2 | Y/P | 4.5 | 7.6 | 14.3 | Ambulatory | NA | 6.8 | NA | exon 70, p.Arg3381* |
p9 | 2 | Y/P | 3 | 8.8 | 14.0 | Ambulatory | NA | 5.3 | NA | exon 33, p.Glu1540* |
p10 | 1.5 | Y/P | 3 | 8.0 | 13.0 | Non-ambulatory | 8.5 | 5.0 | NA | Exon 3, p.Leu1675X |
p11 | n.a | Y/P | 3.5 | 6.9 | 12.2 | Ambulatory | NA | 5.3 | NA | exon 70, p.Arg3381* |