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Selected patients for the study. This table reports the patients in the study, with their specific mutation, D0 and age at the beginning of the treatment. In addition, it reports the gravity of the disease (7 heavily impaired, 1 mildly impaired).
| ID | D0 (Age) | Mutation | Severity (1–7) |
|---|---|---|---|
| S1 | 4.8 | Del exons 3 and 4 | 4 |
| S2 | 8.1 | T158M (missense) | 6 |
| S3 | 5.11 | R133C (missense) | 3 |
| S4 | 5.9 | C13ins in GCCGC in exon 1 (frameshift) | 3 |
| S5 | 4.7 | R270X (nonsense) | 5 |
| S6 | 10.8 | 1055 del 12 + 1157 del 44 (frameshift) | 3 |
