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Evidence found and GRADE certainty

10 questions · 10 findings · 3 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 0 · 0%GRADE not reported 0 · 0%Very low 0 · 0%Low 3 · 30%Moderate 7 · 70%High 0 · 0%

Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis vs on‐demand therapy

All adverse events · people without inhibitors

Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis probably leads to higher all adverse events compared with on‐demand therapy (moderate certainty).

1 finding from 1 review

Summary of findings table ‐ Emicuzimab 3.0 mg/kg bi‐weekly prophylaxis vs on‐demand therapy

All adverse events · people without inhibitors

Summary of findings table ‐ Emicuzimab 3.0 mg/kg bi‐weekly prophylaxis probably leads to higher all adverse events compared with on‐demand therapy (moderate certainty).

1 finding from 1 review

Summary of findings table ‐ Emicizumab 1.5 mg/kg once weekly vs emicizumab 3.0 mg/kg every 2 weeks

All adverse events · people without inhibitors

Summary of findings table ‐ Emicizumab 1.5 mg/kg once weekly showed no clear difference in all adverse events compared with emicizumab 3.0 mg/kg every 2 weeks (moderate certainty).

1 finding from 1 review

Summary of findings table ‐ Concizumab prophylaxis vs on‐demand therapy

Adverse events ‐ All adverse events · people with inhibitors

Summary of findings table ‐ Concizumab prophylaxis showed no clear difference in adverse events ‐ All adverse events compared with on‐demand therapy (low certainty).

1 finding from 1 review

Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo

Treatment‐related AEs: total treatment‐emergent adverse events potentially related to treatment · adults with LOPD

Cipaglucosidase plus miglustat showed no clear difference in treatment‐related AEs: total treatment‐emergent adverse events… compared with alglucosidase alfa plus placebo (low certainty).

1 finding from 1 review

Avalglucosidase alfa vs alglucosidase alfa

Treatment‐related AEs: total treatment‐emergent adverse events potentially related to treatment · adults with LOPD

Avalglucosidase alfa showed no clear difference in treatment‐related AEs: total treatment‐emergent adverse events… compared with alglucosidase alfa (low certainty).

1 finding from 1 review