Table 1 Disease-specific characteristics and treatment overview for all our patients (n = 11)
From: Long term treatment with ataluren—the Swedish experience
Demographics | |
|---|---|
Characteristics | |
Gender, n (%) | |
Male | 11 (100.0) |
Age at symptom onset, years (n = 8; 3:not available) | |
Median | 1.75 |
Min, Max | 1.5, 4.0 |
Age at diagnosis, years (n = 10; 1:not available) | |
Median | 3.75 |
Min, Max | 3.0, 5.0 |
Age at last follow-up, years (n = 11) | |
Median | 16.2 |
Min, Max | 12.2, 26.45 |
Ambulation status at last follow-up, n (%) | |
Ambulatory | 4 (36.4) |
Non-ambulatory | 7 (63.6) |
Age at loss of ambulation, years (n = 7; 4:Not Applicable) | |
Median | 13.2 |
Min, Max | 8.5, 18.1 |
Age at start of ataluren treatment, years (n = 11) | |
Median | 8.4 |
Min, Max | 5.2, 14.4 |
Cumulative use of ataluren, years (n = 11) | |
Median | 6.3 |
Min, Max | 4.0, 9.35 |
Age at start of corticosteroid treatment, years (n = 11) | |
Median | 4.0 |
Min, Max | 3.0, 5.0 |
On-going ataluren treatment at last follow-up, n (%) | 7 (63.6) |
On-going corticosteroid treatment at last follow-up, n (%) | 11 (100.0) |
Age at discontinuation of ataluren treatment, years (n = 4) | |
Median | 17.6 |
Min, Max | 14.2, 25.5 |