What do systematic reviews say?

Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults and children with CF with at least one class I mutation (PTC)

FEV1 % predicted: relative change from baseline · adults and children with CF with at least one class I mutation (PTC)

Adverse events relating to treatment Scale: mild, moderate, severe and total · adults and children with CF with at least one class I mutation (PTC)

Pulmonary exacerbations · adults and children with CF with at least one class I mutation (PTC)

Nutrition and growth Change in: a) weight (kg) b) BMI (kg/m2) c) height (m) · adults and children with CF with at least one class I mutation (PTC)

Sweat chloride level ‐ change from baseline Scale: mmol/L · adults and children with CF with at least one class I mutation (PTC)

Mortality rate · adults and children with CF with at least one class I mutation (PTC)