Table 2.
In vivo response before and during treatment with VX770 (Kalydeco)
| GCST |
Optical sweat bioassay of CFTR functiona |
Lung function |
|||
|---|---|---|---|---|---|
| Weeks of treatment | Treatment | Cl− (mmol/L) | Mean C/M ratio | C glands (n) | FEV1% |
| 0 | off | 107-101/110–112b | 0b | 0b | 64 |
| 1 | on – Kalydeco (VX770) | 36–35 | 0,0119 | 29 | 68 |
| 4 | on – Kalydeco (VX770) | 36–43 | 0,0696 | 81 | NA |
| 12 | on – Kalydeco (VX770) | NA | NA | NA | 65 |
| 20 | on – Kalydeco (VX770) | 23–27 | NA | NA | 68 |
| 27 | on – Kalydeco (VX770) | NA | NA | NA | 70 |
Optical CFTR-dependent sweating test.42
Results obtained at two different times; Sweat Cl− values are reported for both forearms.