Table 4 Evaluation phase results indicating that treatment of nmDMD patients with ataluren, in addition to standard of care, can delay both scoliosis and the decline in patients’ upper limb function, regardless of mobility status
Theme | Statement | Overall Agreement | Consensus |
|---|---|---|---|
Scoliosis | Development of scoliosis has a detrimental impact on patients’ pulmonary function | 100.0% | Very high |
nmDMD patients treated with ataluren (in addition to standard of care) are less likely to develop scoliosis | 85.0% | High | |
Non-ambulatory nmDMD patients are less likely to develop scoliosis if they continue treatment with ataluren (in addition to standard of care) after loss of ambulation | 75.0% | High | |
Upper limb function | Delaying the decline of muscle function in patients’ upper limbs helps to maintain independence | 100.0% | Very high |
Delaying the decline in fine motor skills also enables patients in wheelchairs to continue to be as independent as possible | 100.0% | Very high | |
Delaying the decline of upper limb strength enables non-ambulatory patients to transfer from their wheelchair to the toilet, maintain intimate hygiene, retain independence and protect their quality of life | 100.0% | Very high | |
Decline of upper limb function has a major impact on patients’ quality of life; they become increasingly dependent on others | 100.0% | Very high | |
Ataluren (in addition to standard of care) delays the decline in nmDMD patients’ upper limb function, regardless of mobility status | 95.0% | Very high | |
Ataluren (in addition to standard of care) delays the decline of fine motor skills in nmDMD patients | 95.0% | Very high |