Table 3 Results indicating that there was a high consensus that ambulation in patients should be preserved for as long as possible
Theme | Statement | Overall Agreement | Consensus |
|---|---|---|---|
Loss of ambulation | Ataluren (in addition to standard of care) significantly delays the loss of ambulation in patients with nmDMD | 85.0% | High |
Ataluren (in addition to standard of care) is expected to result in the same treatment effect in each surviving muscle fibre irrespective of the nmDMD patients’ ambulatory status | 65.0% | None | |
If a nmDMD patient receiving ataluren loses ambulation, they should continue treatment with ataluren | 95.0% | Very high | |
There is life beyond loss of ambulation. There are still lots of important functions of the muscles such as being able to use the hands and arms, fine motor skills and respiratory muscles – all these functions should be maintained for as long as possible | 100.0% | Very high | |
Delaying the loss of ambulation in patients with nmDMD may reduce the development of scoliosis | 90.0% | High | |
Delaying the loss of ambulation in patients with nmDMD delays the decline of respiratory function | 100.0% | Very high | |
Delaying the loss of ambulation in patients with nmDMD is related to the decline of upper limb function | 75.0% | High |