Table 5 There was a high or very high consensus regards the importance of delaying loss of pulmonary function and the positive role of ataluren
Theme | Statement | Overall Agreement | Consensus |
|---|---|---|---|
Pulmonary function | Continuing the use of ataluren, in addition to the standard of care, in nmDMD patients when they lose ambulation delays the decline in pulmonary function | 100.0% | Very high |
Maintaining patients’ pulmonary function means they experience fewer respiratory infections and may require less frequent hospitalisations | 100.0% | Very high | |
The ability of nmDMD patients to cough is maintained for longer with ataluren, in addition to standard of care | 95.0% | Very high | |
Ataluren, in addition to standard of care, delays the decline in nmDMD patients’ pulmonary function | 95.0% | Very high | |
Ataluren, in addition to standard of care, significantly delays the decline in nmDMD patients’ pulmonary function | 80.0% | High | |
Ataluren, in addition to standard of care, prolongs nmDMD patients’ ability to breathe independently | 95.0% | Very high | |
Ataluren, in addition to standard of care, significantly prolongs nmDMD patients’ ability to breathe independently | 85.0% | High | |
When nmDMD patients’ FVC falls below 60%, at latest, it becomes necessary to commence physiotherapy and/or screening for night-time ventilation | 85.0% | High | |
Patients that continue to receive ataluren, in addition to standard of care, after loss of ambulation are expected to have a delayed requirement for ventilation | 95.0% | Very high |