Table 6 Clinicians evaluated statements on cardiac function and duration of treatment with ataluren and there was a high or very high consensus across the statements
Theme | Statement | Overall Agreement | Consensus |
|---|---|---|---|
Cardiac function | It is logical to expect ataluren in nmDMD patients to have an effect on all muscles, including the cardiac muscle | 95.0% | Very high |
It is logical to expect ataluren in addition to standard of care, to delay the onset of cardiac decline in patients with nmDMD | 90.0% | High | |
It is logical to expect ataluren, in addition to standard of care, to delay the onset of cardiomyopathy in patients with nmDMD | 90.0% | High | |
It is logical to expect ataluren, in addition to standard of care, to delay the decline in left ventricular ejection fraction with nmDMD | 90.0% | High | |
Duration of treatment with ataluren | It is important to preserve the function of even small muscles in nmDMD patients | 100.0% | Very high |
Treatment with ataluren should be continued as long as both the physician and nmDMD patients are both willing to continue treatment | 90.0% | High | |
As long as nmDMD patients still have functionally important muscles that can be influenced by ataluren they should continue to be treated | 100.0% | Very high |