Disability due to FAP progression Not measured · people with TTR‐FAP
The review found no eligible studies for this question.
1 finding from 1 review
no estimate extracted
Studies not reported
No studies found
Severity of peripheral neuropathy (expressed as the change in mNIS+7Ionis) Scale from: ‐22.3 (normal) to 346.3 (total impairment) · people with TTR‐FAP
1 finding from 1 review
QoL · people with TTR‐FAP
1 finding from 1 review
Has died · people with TTR‐FAP
Inotersen showed no clear difference in number of participants who died during the trial compared with placebo (low certainty).
1 finding from 1 review
Withdrawals due to adverse events · people with TTR‐FAP
Inotersen may leads to higher number of dropouts due to adverse events compared with placebo (low certainty).
1 finding from 1 review
Number of participants experiencing at least one severe adverse event · people with TTR‐FAP
Inotersen showed no clear difference in number of participants experiencing at least one severe adverse event compared with placebo (low certainty).
1 finding from 1 review
Muscle strength · participants with Lambert‐Eaton myasthenic syndrome
1 finding from 1 review
Improvement in mean CMAP amplitude (mV) · participants with Lambert‐Eaton myasthenic syndrome
1 finding from 1 review
Adverse events · participants with Lambert‐Eaton myasthenic syndrome
1 finding from 1 review
Short‐term (1 week) percentage change (improvement) in overall speech production · people with speech disorder resulting from hereditary ataxias
1 finding from 1 review
Short‐term (1 week) change in isolated movement, objective and subjective measures of speech production SARA. Scale from: 0 to 6. Higher scores indicate more severe speech disorder. · people with speech disorder resulting from hereditary ataxias
1 finding from 1 review
Short‐term (within 1 month) change in quality of life scores related to communication as measured by validated communication assessments · people with speech disorder resulting from hereditary ataxias
1 finding from 1 review
Longer‐term (minimum 1 month) change in generic quality of life scores · people with speech disorder resulting from hereditary ataxias
1 finding from 1 review
Adverse effects (during study) · people with speech disorder resulting from hereditary ataxias
1 finding from 1 review
Longer‐term burdens (minimum 1 month) (for example, demands on caregivers, frequency of tests, restrictions on lifestyle) · people with speech disorder resulting from hereditary ataxias
1 finding from 1 review
Economic outcomes · people with speech disorder resulting from hereditary ataxias
1 finding from 1 review
Cardiac function · patients with established cardiomyopathy in DMD
1 finding from 1 review
Size of metabolically abnormal areas of myocardium · patients with established cardiomyopathy in DMD
The review found no eligible studies for this question.
1 finding from 1 review
no estimate extracted
Studies not reported
No studies found
Improvements in quality of life measures · patients with established cardiomyopathy in DMD
The review found no eligible studies for this question.
1 finding from 1 review
no estimate extracted
Studies not reported
No studies found
Adverse events · patients with established cardiomyopathy in DMD
1 finding from 1 review